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  • Title: Progeria Research Foundation | Home
    Descriptive info: .. About Progeria.. Progeria 101/FAQ.. The Connection to Other Diseases.. The Science Behind Progeria.. The FTI Drug.. For School Reports.. About PRF.. Our Story.. Our Accomplishments.. The People.. PRF By The Numbers.. Financial Profile.. Our Brochure and Logo.. Quick Facts.. Chapters.. Progeria en espanol.. Meet the Kids.. Kids Gallery.. In Memory Of.. NHRA Champion Erica Enders with Zach!.. Zach's Video.. Cam's Video.. Ashley's Video.. Interview with John Tacket.. Medical Research.. What's New in Progeria Research.. Clinical Trials.. Medical Database.. Cell Tissue Bank.. Diagnostic Testing.. Research Funding Opportunities.. Scientific Meetings.. Scientific Publications.. Grand Rounds.. Workshop 2010 Videos.. Parents Doctors.. International Progeria Registry.. Patient Care/Treatments.. Our Programs.. News.. News 2014.. TEDx Talks.. News Archive.. Press Room.. Public Service Announcements.. Newsletters.. Get Involved.. Hats ON for Progeria.. Be a Miracle Maker.. Shop in Our Store.. Sell on eBay.. goodsearch.. com and goodshop.. com.. Mailing List Signup.. Translators Needed.. Fundraising Events.. Events.. Recent Events 2014.. Past Events 2013.. Past Events 2012.. Past Events 2011.. Past Events 2010.. Past Events.. Contact Us.. Donate Now.. Join Our Circle of Hope.. Donate By Credit Card Online.. Other Ways to Support PRF.. MISSION: To discover treatments and the cure for Progeria and its aging-related disorders.. Find out how you can help.. Join Our Community.. Your support keeps us focused on our mission to find a cure.. Featured Video..  ...   development, this first-ever study to assess whether treatments influence patient survival shows that drugs tested in PRF-funded clinical trials increase estimated lifespan of children with Progeria by at least 1.. 6 years.. Click.. here.. to view this exciting study.. March 2014.. PRF On the Move: Receives Research!America Award, Achieves Highest 4-Star Rating.. Recognized in Washington DC for its “significant impact in advancing research”, and attaining the top score from the premier nonprofit overseer, PRF is thrilled to be recognized in such significant ways.. September 24, 2012.. WE DID IT! First-ever Treatment for Progeria Discovered!!.. History has been made, with every child in the first-ever Progeria clinical drug trial showing improvement in one or more areas of their condition, proving that the FTI drug lonafarnib is the first known, effective treatment for children with Progeria.. August 9, 2014 in Brownstown, IN:.. Zach Pickard Pelican Run.. August 17, 2014 in Falmouth, MA:.. Team PRF runs the Falmouth Road Race- Donate Now!.. September 6, 2014 in Peabody, MA:.. 13th Annual International Race for Research.. Dedicated to Sam Berns.. (Register TODAY).. September 26, 2014 in Maumee, OH:.. Carly’s Party.. This site was last updated on September 02, 2014.. Quick Links.. Translate.. Powered by.. Translation Feature Disclaimer.. Home.. Site Map.. Links.. RSS.. Copyright, Privacy Photo Use Policy.. Site by Useful Studios.. 2014.. All Rights Reserved..

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  • Title: Progeria Research Foundation | About Progeria
    Descriptive info: "Isolating the Progeria gene is a major achievement for the medical research community," said Dr.. Collins, "The discovery not only gives hope to children and families affected by Progeria, but also may shed light on the phenomenon of aging and cardiovascular disease.. ".. Hutchinson-Gilford Progeria Syndrome ("Progeria", or "HGPS") is a rare, fatal genetic condition characterized by an appearance of accelerated aging in children.. Its name is derived from the Greek and means "prematurely old.. " While there are different forms of Progeria*, the classic type is Hutchinson-Gilford Progeria Syndrome, which was named after the doctors who first described it in England; in 1886 by Dr.. Jonathan Hutchinson and in 1897 by Dr.. Hastings Gilford.. HGPS is caused by a mutation in the gene called  ...   lead to the process of premature aging in Progeria.. Although they are born looking healthy, children with Progeria begin to display many characteristics of accelerated aging at around 18-24 months of age.. Progeria signs include growth failure, loss of body fat and hair, aged-looking skin, stiffness of joints, hip dislocation, generalized atherosclerosis, cardiovascular (heart) disease and stroke.. The children have a remarkably similar appearance, despite differing ethnic backgrounds.. Children with Progeria die of atherosclerosis (heart disease) at an average age of fourteen years.. * Other progeroid syndromes include Werner's syndrome, also known as "adult progeria" which does not have an onset until the late teen years, with a life span into the 40's and 50's.. Click here.. for information on scientific articles on Progeria.. quick links..

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  • Title: Progeria Research Foundation | Progeria 101/FAQ
    Descriptive info: What is Progeria?.. Hutchinson-Gilford Progeria Syndrome Progeria or HGPS is a rare, fatal genetic condition characterized by an appearance of accelerated aging in children*.. Its name is derived from Greek and means "prematurely old.. " While there are different forms of Progeria.. *.. , the classic type is Hutchinson-Gilford Progeria Syndrome, which was named after the doctors who first described it in England: in 1886 by Dr.. Jonathan Hutchinson, and in 1897 by Dr.. Other Progeroid syndromes include Werner s syndrome, also known as adult progeria which does not have an onset until the late teen years, with a lifespan into the 40 s and 50 s.. How common is Progeria?.. Progeria affects approximately 1 in 4 - 8 million newborns.. There are an estimated 200-250 children living with Progeria worldwide at any one time.. It affects both sexes equally and all races.. Since The Progeria Research Foundation was created in 1999, we have discovered children with Progeria living in over 40 countries.. For a map of where living children reside, please go to our.. page.. What are the features of Progeria?.. Although they are usually born looking healthy, most children with Progeria begin to display many characteristics of Progeria within the first year of life.. Progeria signs include growth failure, loss of body fat and hair, aged-looking skin and stiffness of joints.. As children get older, they suffer from osteoporosis, generalized atherosclerosis, cardiovascular (heart) disease and stroke.. The children have a remarkably similar appearance despite differing ethnic backgrounds.. Children with Progeria die of atherosclerosis (heart attacks or strokes) at an average age of thirteen years.. What is the cause of Progeria?.. HGPS is caused by a mutation in the gene called.. LMNA.. (pronounced lamin-a ).. The.. gene produces the lamin A protein which is the structural scaffolding that holds the nucleus of a cell together.. The abnormal lamin A protein that causes Progeria is called.. progerin.. Progerin makes the nucleus unstable.. That cellular instability leads to the process of premature aging and disease in Progeria.. Progeria Cell Nucleus.. PRF was the driving force behind finding the gene responsible for Progeria.. A group of leading scientists from PRF s Genetics Consortium was able to isolate the Progeria gene, and in April 2003, PRF led the announcement reported in the top scientific journal.. Nature.. v.. Isolating the Progeria gene is a major achievement for the medical research community,.. said Dr.. Francis Collins, Director of the National Institutes of Health and senior discoverer of the Progeria gene,.. The discovery not only gives hope to children and families affected by.. Progeria, but also may shed light on the phenomenon of aging and cardiovascular disease.. Recurrent de novo point mutations in lamin A cause Hutchinson-Gilford Progeria Syndrome ,.. , Vol.. 423, May 15, 2003.. What does Progeria have to do with aging?.. We now know that there is a biological overlap between children with Progeria and the general aging population.. We all make a little bit of.. , the disease-causing protein in Progeria.. We make much less progerin than children with Progeria, but the progerin builds up over a lifetime and may be partly responsible for aspects of aging such as atherosclerosis.. Progerin is also linked to telomere dysfunction.. Telomeres are proteins that play a major role in cellular aging.. Children with Progeria are genetically predisposed to premature, progressive heart disease.. Death occurs almost exclusively due to widespread.. heart disease, the leading cause of death worldwide..  ...   appearance and medical records), a sample of the child s blood will be tested for Progeria.. Now there is a definitive, scientific way to diagnose the children.. This leads to more accurate and earlier diagnoses so the children can receive proper care.. What is PRF doing to help children with Progeria?.. The Progeria Research Foundation funds medical research aimed at developing treatments and a cure for Progeria.. PRF also has its own.. Cell and Tissue Bank.. that provides the biological materials researchers need to conduct their experiments.. Additionally, PRF has established a.. Medical and Research Database.. to supply physicians and families with medical recommendations for cardiac care, nutrition and other medical issues to help children and adults with Progeria have a better quality of life.. We continue to analyze medical records of children and adults with Progeria so that we can provide information on how best to medically help those with Progeria, and provide clues towards potential new treatments.. In April 2010, PRF published.. The Progeria Handbook.. for families and doctors.. From basic health facts to daily care recommendations to extensive treatment guidelines, the handbook helps answer many questions for people suffering from Progeria throughout the world.. The Handbook is available in English, Spanish and Portuguese.. PRF is also driving Progeria clinical drug trials testing potential treatments.. To date, PRF has funded and co-coordinated three clinical trials.. Please refer to.. clinical drug trials.. for more details.. History was made in September 2012 with the discovery that the drug involved in the first clinical trial,.. a farnesyltransferase inhibitor or FTI.. , is.. the first-ever treatment for Progeria.. PRF continues to explore other promising drugs that will bring us to a cure for Progeria.. Proc Natl Acad Sci U S A.. 2012 Sep 24.. [Epub ahead of print].. What can you do to help children with Progeria?.. Make a financial contribution.. Donations are needed to continue the vital work of PRF.. No donation is too little or too big every dollar counts in our quest for a cure! Online donations are accepted on our.. Ways to Donate.. Donate your time.. Volunteers are also important to PRF s success.. Hold a special event like a bake sale or letter writing campaign; translate documents for the families; help with a mailing we ll find something for you to do that fits your schedule, location and talents! Visit our.. page to see all the ways you can help.. Donate in-kind services or items.. Do you own a printing or office supply business? Are you a computer whiz that can help with our.. Twitter.. ,.. Facebook.. , web site and/or other on-line presence? Do you have a business background in non-profit development? These are just some of the many types of talents and connections PRF needs.. The more tasks we can get accomplished on a pro bono basis, the more money we can devote to research!.. Spread the word and tap into your connections.. Do you know anyone who can help?.. Tell your friends, family and colleagues about PRF and the amazing work we re doing! Many times, people are more likely to donate to an organization if they ve gotten a recommendation from someone they know.. So put in a good word for us and tell everyone to visit our web site at.. www.. progeriaresearch.. org.. , follow us on.. , like us on.. , and contact us at.. info@progeriaresearch.. org.. TOGETHER WE.. WILL.. FIND THE CURE!..

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  • Title: Progeria Research Foundation | The Connection to Other Diseases
    Descriptive info: Aging.. heart disease, the leading cause of death in the US, and #2 worldwide.. As with any person suffering from heart disease, the common events for Progeria children are strokes, high blood pressure, angina, enlarged heart, and heart failure, all conditions associated with aging.. "These children get heart disease at an incredibly accelerated pace, usually by the time they are 12, 13 or 14.. In the general community, the prevalence of cardiovascular disease starts appearing in the 60s and 70s.. Obviously, there's some process that's accelerated.. ".. Dr.. Samer Najjar, head of the Human Cardiovascular Studies Unit at the National Institute on Aging.. Thus there is clearly a tremendous need for research in Progeria.. Because.. finding a cure for Progeria will not only help these children, but may provide keys for treating millions of adults with heart disease and stroke associated with the natural aging process.. Because the aging process is accelerated in children with Progeria, they offer researchers a rare opportunity to observe in just a few years what would otherwise require decades of longitudinal studies.. ".. A better understanding of the causes of this syndrome (Progeria) could lead to better insights into the mechanisms of both development and  ...   as heart disease).. Atherosclerosis is just one of several types of "arterio"-sclerosis, which is characterized by thickening and hardening of the arteries, but the two terms are often used to mean the same thing.. Some hardening of the arteries often occurs when people grow older.. Atherosclerosis involves deposits of fatty substances in the inner lining of an artery.. This buildup is called plaque.. Plaques can grow large enough to significantly reduce the flow of blood through an artery, or plaques become fragile and rupture to form clots that can block blood flow.. If the blockage occurs to an artery that feeds into the heart, it causes a.. heart attack.. If the blockage occurs to an artery that feeds into the brain, it causes a.. stroke.. Atherosclerosis is not only the cause of death among children with Progeria, but also the.. leading cause of death in the United States and many other parts of the world in normal aging individuals.. It is our hope that the discovery of the Progeria gene will have a beneficial effect not only for Progeria patients, but also for the millions of people affected by aging-related conditions, including heart attacks and stroke caused by atherosclerosis and arteriosclerosis..

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  • Title: Progeria Research Foundation | The Science Behind Progeria
    Descriptive info: As with any person suffering from heart disease, the common events for Progeria children are high blood pressure, strokes, angina (chest pain due to poor blood flow to the heart itself), enlarged heart, and heart failure, all conditions associated with aging.. Hutchinson-Gilford Progeria Syndrome ("Progeria", or "HGPS") is caused by a mutation in the gene called LMNA (pronounced, lamin - a).. A group of leading scientists from The Progeria Research Foundation's Genetics Consortium was able to isolate the Progeria gene in October 2002, and in April 2003, PRF led the announcement that Progeria is caused by a mutation of the gene LMNA, or Lamin A.. This gene discovery was reported in the leading scientific journal Nature.. The Progeria gene finding involved intensive collaboration  ...   at University of Michigan's Department of Human Genetics, Dr.. Francis Collins, Director of the National Human Genome Research Institute (responsible for mapping the human genome) and the senior author on the report, and first author Dr.. Maria Eriksson, a postdoctoral fellow with Dr.. Collins.. How is Progeria Diagnosed?.. Now that the gene mutation has been identified,.. The Progeria Research Foundation has created a.. Diagnostics Testing Program.. After an initial clinical evaluation (looking at the child's appearance and medical records), a sample of the child's blood will be tested for the Progeria gene.. For the first time ever, there is a definitive, scientific way to diagnose the children.. This will lead to more accurate and earlier diagnoses so that the children can receive proper care..

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  • Title: Progeria Research Foundation | The FTI Drug
    Descriptive info: FTIs - a potential drug treatment for children with Progeria.. In August 2005 and February 2006, researchers published studies that support a potential drug treatment for children with Progeria.. Farnesyltransferase inhibitors (FTIs).. , originally developed for cancer, are capable of reversing the dramatic nuclear structure abnormalities that are the hallmark of  ...   some signs of disease in a Progeria-like mouse model.. Here are some answers to.. Frequently Asked Questions about FTIs.. and these exciting findings.. Did You Know?.. Progeria is caused by a tiny point mutation in a child's DNA, a one-letter typo in the billions of letters that make up the chromosomal book..

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  • Title: Progeria Research Foundation | For School Reports
    Descriptive info: “My fifteen-year-old daughter just wrote a research paper on this topic, and she was so moved by the precious children, that she asked to donate her own money to this cause.. It's great that you have things on the web that can give these children exposure and a voice to those of us who may never personally meet one!”.. Lisa Hagen.. Do you have questions you need answered for a school report? Do you want to learn more about the children? Are you interested in knowing more about Progeria and the work PRF does? We've got the answers for you right here!.. We love the fact that awareness about Progeria is spreading throughout the world and that you want to learn more, thank you! But we are so very busy at the office doing all the things that are needed to advance our mission of finding treatments and a cure that.. we can no longer individually answer the many requests we receive from around the world.. Instead, we have prepared this Question and Answer sheet to help provide you with the information you're looking for, taking the most common questions we are asked and answering them below.. We hope this helps you get top grades on your school report, or just helps you understand more about Progeria and The Progeria Research Foundation so that you can help spread the word about the wonderful work we are doing for these special children.. 1.. I'm doing a school report on Progeria, can you help me get more information on this subject?.. 2.. I heard that the Progeria gene had been discovered, where can I find out more about this?.. 3.. Is Progeria a dominant or recessive disease?.. 4.. I need to interview a researcher or other expert in the field of Progeria.. 5.. How many children in the world are diagnosed with Progeria?.. 6.. Where do the children live?.. 7.. Do the children participate in school activities, or are they limited because they have Progeria?.. 8.. What is it like for a child to live with Progeria?.. 9.. Does the disease affect their mental abilities?.. 10.. Why do children with Progeria age in body so rapidly and not in mind?.. 11.. What issues do you think are most important in the care of a patient with Progeria?.. 12.. Are there any treatments available?.. 13.. Are any scientists close to finding a cure?.. 14.. What research are you currently doing on the disease?.. 15.. I would like permission to use some of the photos and information from your website for my project.. 16.. I saw HBO Film s.. and/or a program on TV and would like a copy of the tape.. 17.. What would you like people to know about Progeria now?.. 18.. I would like to study Progeria for a career, what courses of study do you suggest I take?.. 19.. I'd like to be a pen pal to a child with Progeria or send them a present.. 20.. I would like to talk to someone by phone or in person about living with Progeria.. 21.. I'd like to volunteer to work with the children.. Do you have a camp that you run, or is there a hospital that I can contact regarding working with children with Progeria?.. 22.. What can people do to help? Where do you get most of your funding?.. 23.. Is there a chapter near where I live that I can contact?.. I'm doing a school report on Progeria, can you help me get more information on this subject?.. In addition to finding the cure and effective treatments for Hutchinson-Gilford Progeria Syndrome, part of our mission is to promote public awareness of this disease.. By doing a report on Progeria, you are helping us accomplish that part of our mission.. Thanks so much, we're glad to help you!.. Before you read any further, please visit our.. Frequently Asked Questions.. section to learn more about Progeria.. Other sections you may find helpful are.. , and.. The information on those pages is not repeated here.. If you are looking for more advanced, scientific information, please visit.. http://www.. pubmed.. gov/.. which provides abstracts for many scientific publications on Progeria.. For key scientific publications that have helped advance Progeria research since the 2003 Progeria gene discovery, visit our.. What s News in Progeria Research.. section.. Also, you can access information on the NORD (National Organization of Rare Diseases) web site.. Go to.. rarediseases.. org/.. , and click on their Rare Diseases Database, then type Hutchinson Gilford Progeria in the search box, and a page on Progeria will appear.. If you want a detailed report, there may be a cost.. For books on Progeria, here are a few publications that you may find useful:.. Keith Moore, father of a child who died of Progeria, wrote.. Old at Age 3.. , the story of Zachary Moore.. ,.. to share the story of his son s extraordinary life.. Leslie Gordon, PRF s Medical Director, wrote a chapter on Progeria for the.. World Book Online Reference Center.. worldbookonline.. com/wb/Article?id=ar447180.. and the print edition of the 2008 World Book Encyclopedia.. Gordon, W.. Ted Brown and Frank Rothman wrote a chapter entitled.. LMNA and the Hutchinson-Gilford Progeria Syndrome and Associated Laminopathies.. for the book.. Inborn Errors of Development: The molecular basis of clinical disorders of morphogenesis.. (2007, 2nd ed.. ) 139: 1219-1229.. If you give us your address, we can send you a.. brochure.. and our.. latest newsletter.. to include with your report.. Back to Top.. I heard that the Progeria gene  ...   2012 in.. Proceedings of the National Academy of Sciences.. Further studies published in May 2014.. reveal that lonafarnib also increases the children s estimated lifespan.. We now know that drugs can improve the disease, as we continue to search for even more effective compounds and ultimately, the cure.. The Progeria Research Foundation helped in the discovery of the gene that causes Progeria, and is now involved in.. clinical drug trials,.. testing drugs that show great promise to effectively treat children with Progeria.. We have made tremendous progress, but no one can predict how long that will take.. What research are you currently doing on the disease?.. See our.. Grants we have Funded.. section for project descriptions, our.. updates and.. PRF In The News.. for the latest research findings.. It is fine to use any text information gathered from our website for your project.. However, we would prefer that you not use photos of the children from our web site.. We suggest you use our.. brochure.. and newsletter, which have lots of photos of children with Progeria, as visuals.. If you give us your address, we can send you one.. You can now buy the Life According to Sam DVD from the.. HBO store.. Please also check out our.. LATS page.. and learn more about this wonderful film and riveting documentary about love, life and hope for children with Progeria.. The Progeria Research Foundation does not own videotapes of shows about Progeria.. These documentaries and other videos are privately produced and due to copyright restrictions, cannot be sold by private organizations or individuals.. You may want to contact the network on which you saw the piece; they might provide you with one for a fee.. In addition, some shows are on line, like the.. Katie Show.. You should also check out two inspiring TEDx Talks by Dr.. Leslie Gordon, our Medical Director, and Sam Berns on our.. Tedx Talks page.. Finally, we invite you to visit our.. YouTube.. page for dozens of informative and inspiring videos.. What would you like people to know about Progeria now?.. While Progeria is an extremely rare disease, all children with Progeria die of severe premature atherosclerosis (heart disease).. When you help children with Progeria, you are helping to save the lives of children with a 100% fatal disease.. At the same time, you are helping us all, because research in this field will not only help The Progeria Research Foundation toward our goal of finding a treatment and a cure, but may provide answers to the mysteries of the natural aging process, heart disease and stroke (one of the leading killers in the world).. I would like to study Progeria for a career, what courses of study do you suggest I take?.. Wow - that's terrific! Depending on where you are in your education, this answer varies.. We suggest that you consult with your guidance or career counselor at school for the best course of action.. Your idea of wanting to become pen-pals is really great, but all contact information for the families and the children is confidential, so we wouldn't be able to give you their names and email addresses.. However, the.. links.. section lists some web sites for families with children that have Progeria.. You can contact them through their web sites and ask if they'd like to have a pen-pal or a gift.. For the same confidentiality reasons as described in the answer above, we cannot put you in touch with someone to talk about living with Progeria.. Please refer to #8 above to get information on this subject.. Do you have a camp that you run, or is there a hospital that I can contact regarding working with children with Progeria?.. We do not have programs where we have direct contact with the children in the way that you are thinking of.. The children are located all over the world, and so our contact with the families is almost exclusively through email, phone and postal mail.. There is no "camp" or other social meeting we are involved in that brings them together where you might have an opportunity to work with the children.. But we can always use another volunteer on our team! Go to the.. Other Ways to Help.. section for more information.. The Progeria Research Foundation has been able to do a tremendous amount with a small amount of money.. We don't have an endowment fund, and rely on the continued support of others who support our mission.. Every little bit helps - those ten and twenty dollar donations really add up! Please check out our.. Miracle Makers.. for just some of the great ways people have become involved, like a group of high school students who produced a performance, a French policeman who organized a flea market and a local bank that held a casual day - all to raise funds for PRF.. Every donation counts, and will allow us to work faster towards a cure, to win this race against time for all children with Progeria throughout the world.. The best ways people can help is by making a donation, organizing a fundraiser and getting others to donate.. Click here to Donate Now!.. PRF now has several chapters in the United States! Visit our.. Chapters section.. to support a chapter in your area, and read all about the wonderful work these groups are doing to help in our quest for a cure.. We'd love for you to join our chapters in any way you can - Together, we.. find the cure!..

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  • Title: Progeria Research Foundation | About Progeria Research Foundation
    Descriptive info: About Progeria Research Foundation.. With the exception of our staff, everyone involved with PRF is a volunteer! Our Board of Directors, Clerk, Treasurer, committee members, translators, fund-raisers, etc.. all devote their time, energy and talents to furthering our mission without pay.. The MISSION of The Progeria Research Foundation:.. To discover treatments and the cure for Progeria and its aging-related disorders.. September 2012.. Progeria is a rare and fatal genetic disease of accelerated aging in children.. Without the discovery of new treatments, all children with Progeria will die of heart disease at an average age of 14 years.. The Progeria Research Foundation (PRF) was founded in 1999 in response to the complete lack of progress being made to help children with Progeria.. Our original mission: to discover the cause, treatments and cure for Progeria.. * Today, PRF continues to be the.. only.. organization in the world solely dedicated to this mission.. We have filled a void, taking these children out of the background where they had been for over 100 years and putting them and Progeria at the forefront of scientific efforts.. In just 13 years, we have achieved extraordinary progress towards our mission: the Progeria gene discovery in 2003, first-ever clinical drug trials initiated in 2007, and the 2012 results of that first trial being the discovery of the first treatment for Progeria.. In addition, we have achieved extensive global awareness of the disease and PRF's work, and confirmation of critical biological links between Progeria, heart disease and aging we  ...   and cure.. And along the way, we are learning a tremendous amount about ourselves.. Please enjoy the pages of this web site, which details PRF's programs, progress and partners.. Most recently, the historic Progeria treatment discovery, tremendous success of our global campaign to identify all children with Progeria, and plans for continued clinical drug trials and drug discovery are contributing to an exciting 2012.. All of these initiatives will continue to be strong in 2013, along with the many other activities which have edged us ever-closer to our ultimate goal of a cure.. Your love and support for children with Progeria makes these exciting strides possible.. Audrey and her nephew Sam.. celebrate Night of Wonder 2009.. Onward and Upward!.. Audrey Gordon.. President, Executive Director.. agordon@progeriaresearch.. *In 2004, in response to the Progeria gene discovery and the growing scientific evidence linking Progeria to the heart disease and aging that affects us all, the mission was revised:.. To discover treatments and the cure for Progeria and its aging related disorders.. **.. Most of the time, medical research is like a marathon, but researchers today published the result of a.. scientific sprint.. the first treatment for Progeria,.. when ten years ago the cause was not known.. With.. an unexpected payoff for the rest of the humanity.. for other diseases associated with aging.. -.. Reporters Robert Siegel and Jon Hamilton, on National Public Radio s.. All Things Considered.. airing September 24, 2012, the day PRF announced the discovery of the first-ever treatment for Progeria..

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  • Title: Progeria Research Foundation | Our Story
    Descriptive info: How PRF was formed.. In the summer of 1998, Dr.. Leslie Gordon and Dr.. Scott Berns found out that their son Sam, who was then 22 months old, had been diagnosed with Hutchinson-Gilford Progeria Syndrome ("Progeria"), commonly referred to as a "premature aging" syndrome.. It quickly became apparent to Sam's parents that there was an enormous lack of medical information and resources dedicated to Progeria.. They recognized that there was no place for these children to go for medical help, no place for parents or doctors to turn for information, and no source of funding for researchers who wanted to do Progeria research.. The lack of information available to families, combined with the lack of research and research-funding opportunities inspired Sam's family, together with their friends and colleagues, to launch The Progeria Research Foundation, Inc.. ("PRF"), the only non-profit organization in the world dedicated to Progeria research.. Sam passed away on January 10, 2014, leaving a legacy of inspiration that now drives PRF and its supporters to continue the quest for a cure, with more determination than ever.. to  ...   this syndrome.. Since its inception, PRF has benefited from the leadership of Attorney Audrey Gordon, Sam's aunt, who serves as the organization's President and Executive Director.. Did you know?.. As a result, our administrative costs are very low.. This leaves more money to devote to medical research and raising public awareness, which ultimately lead to finding a cure for Progeria.. Leslie B.. Gordon, MD, PhD, is PRF's Medical Director.. She is also the Principal Investigator of PRF's research-related programs: The PRF International Registry, Cell Tissue Bank, Medical Research Database, and Diagnostics Testing Program, and co-author on the historic Progeria gene finding and treatment discovery*.. * Thanks to PRF's efforts, in April 2003 PRF and the National Institutes of Health announced that.. the cause of Progeria, a mutation in the LMNA gene, was found.. , and in September 2012,.. the first-ever treatment was discovered.. There is much work to be done and little resources with which to do so.. We cannot do it alone.. With your support, the cure will be discovered for these wonderful children.. Together, we.. find the cure..

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  • Title: Progeria Research Foundation | Progeria Research Foundation's Accomplishments
    Descriptive info: Progeria Research Foundation's Accomplishments.. Promotion of Education and Awareness.. History, Accomplishments Goals (updated September 2012).. In just 13 years, since we founded PRF and there were no resources for these children, we have gone from gene finding to the first clinical trials in Progeria, to a treatment.. We are hailed as a model for disease-research organizations and a prime example of a successful translational research organization, moving from the lab to treatments at a pace virtually unheard of in the scientific community.. And while helping this handful of children, the connection of Progeria to common heart disease and aging has tremendous implications for us all.. Click here to view our Timeline Highlights from 1999 through September 2012!.. PRF's Annual Report 2011.. PRF's Annual Report 2010.. PRF's 2009 End of Year Report.. The Progeria Research Foundation, Inc.. (PRF) was founded in March 1999 by the family, friends and colleagues of Sam, a Massachusetts child with Progeria.. Progeria is a fatal, rapid aging disease that afflicts children, who die of heart disease at an average age of 14 years.. VISION:.. To be the driving force worldwide to:.. Discover the cure.. Develop treatments.. Provide information and guidance.. Be a valuable resource for Progeria families.. The governance of the corporation is vested in a national Board of Directors which serves without compensation and meets quarterly.. There is also a volunteer Medical Director and corporate officers.. Volunteer committees (public awareness, medical research, special events and development committees) meet as needed.. Staff includes an Executive Director, Clinical Trial Coordinator, Director of Volunteer Activities, Donor and Communications Specialist, and Administrative Assistant.. Working with PRF are also a part-time grant writer and major gifts officer.. PRF is the only organization in the world solely dedicated to finding a cure for Progeria.. We have progressed rapidly by discovering the Progeria gene in 2003, starting clinical drug trials just 4 years later, and discovering the first treatment in 2012! And finding a cure will help not only those with Progeria, but perhaps also the millions of people who suffer from heart disease and other, aging-related conditions.. PRF S ACCOMPLISHMENTS.. In the first few years of existence, PRF created a comprehensive network of research-related needs to promote advances in the field, such as a cell tissue bank, diagnostic testing, international registry, grant funding and scientific workshops.. By centralizing all of the resources needed to propel forward as quickly as possible towards treatments and cure, PRF has firmly established itself as an international force driving Progeria research and education.. The following PRF activities have played and are playing key roles in Progeria research:.. Discovery of the Progeria Gene:.. PRF was instrumental in this historic, 2003 accomplishment! Members of PRF s Genetics Consortium collaborated, and cell lines from PRF s Cell Bank were essential to the experiments that led to the discovery - a critical step in the ultimate quest for a cure.. The gene discovery literally flung open the doors of science, and all of our programs have grown, as the number of potential patients coming to PRF for diagnosis and treatment, and the number of scientists interested in Progeria research, rises - all because of our accomplishments and outreach efforts.. Diagnostics Testing Program:.. PRF created this project in the wake of the gene discovery so that children, their families and medical caretakers can for the first time be given a definitive, scientific diagnosis.. This can translate into earlier diagnosis, fewer misdiagnoses and early medical intervention to ensure a better quality of life for the children.. In addition, because this definitive diagnostic testing prevents misdiagnosis, it ensures that scientists are working with the proper cells and tissues, as they ask questions about Progeria, heart disease and aging.. First-Ever Progeria Clinical Drug Trial for Progeria:.. PRF funded and co-coordinated the first-ever clinical drug trial for Progeria, taking place at Children s Hospital Boston.. The trial drug is called a farnesyltransferase inhibitor, or FTI, a drug that has shown great promise in the laboratory and in animal models.. The first trial began in May 2007, involved 28 children (ages 3 to 15 years) from 16  ...   basic health facts to daily care recommendations to extensive treatment guidelines, this 100-page handbook helps answer many questions for children with Progeria throughout the world, and is available in English, Spanish and Portuguese.. The database was critical in determining the primary clinical outcome parameter for the first-ever clinical drug trial for Progeria.. Scientific Workshops on Progeria:.. PRF has organized six successful scientific conferences that have brought together scientists and clinicians from all over the world to collaborate, sharing their ideas and contributing their expertise in this lethal disease.. The most recent workshop.. , "From Bench to Bedside in a Decade," took place April 11-14, 2010 in Boston, MA with 140 participants from ten countries.. Highlights included a.. plenary address.. given by PRF's Medical Director and a.. Progeria family panel.. The next workshop is scheduled for April 2013 in Bethesda, MD.. Research Grants:.. PRF s grants of up to $100,000 per year, for up to three years have allowed innovative research in Progeria to thrive.. Proposals are carefully evaluated by PRF s Medical Research Committee and Board of Directors.. We know more about Progeria and its connection to heart disease and aging than ever before, and we re on our way to a future of better health and longer lives for the children.. To date, PRF has invested over $5 million to fund 54 grants for Progeria-related research projects performed in 17 states and 10 other countries.. Publication of Scientific Papers:.. A major goal of PRF is to promote awareness about Progeria and the progress being made in the field of Progeria research.. Dozens of publications on Progeria, many of which acknowledge PRF grant support or PRF s support of providing cells, have been published in well-known, respected scientific journals read by thousands of researchers worldwide.. From 2002-2011, 272 scientific publications have appeared, compared to less than 2 per year for the previous 50 years - that's over a 1,500% average annual increase!.. Web Site/Public Awareness:.. ProgeriaResearch.. org provides access to the latest information on Progeria research, support, and education for families and caregivers.. PRF s newsletters reach over 17,000 people in 61 countries.. PRF s story has appeared on CNN, The Dr.. Oz Show, Primetime Live, Dateline, and The Today Show, and in.. Time.. and.. People.. magazines,.. The New York Times.. The Wall Street Journal.. (front page!) and dozens of other widely-read media outlets.. In October 2009, PRF and its partner.. GlobalHealthPR.. launched a global awareness campaign called.. Find the Other 150.. to drive the search for unidentified children with Progeria worldwide.. The campaign has helped PRF to discover new families and children with Progeria who need our help.. findtheother150.. Volunteer Expansion:.. Thousands have joined in PRF s fight for a cure in many ways, from participating in fundraisers, to translating information for the non-English speaking families, to stuffing envelopes! The.. section of PRF s newsletters and web site pay tribute to these heroes who selflessly give their time talents to raise money and awareness for PRF.. In 2005, we created our first.. chapter.. , in Southern California, and six more have been formed.. PRF in 2012 and Beyond.. In the years to come, we intend to live up to our reputation of rapid and impactful accomplishments, including the following:.. Work towards.. new treatments and treatment trials.. for children with Progeria.. Continue our efforts to.. promote.. drug discovery.. and partner with drug companies, to develop potential treatments and the cure for Progeria.. Continue to.. expand the.. Find the Other 150.. Campaign.. globally, to increase awareness of Progeria so more children will be found and helped by PRF.. Increase.. the number of participants in.. the International.. Registry.. and.. Medical Research.. Database.. projects, so that scientists can continue to use them as research tools to understand Progeria, heart disease and aging.. Fund.. additional.. research.. proposals.. that focus on studying the Progeria gene and how this defect can be corrected.. engage.. motivate.. our enthusiastic.. volunteer community.. to further increase revenue and expand our programs.. back to top.. was just completed in December 2009.. Trial results will be published in a scientific journal in.. near future..

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  • Title: Progeria Research Foundation | The People
    Descriptive info: Officers and Staff.. Volunteer Board.. Committees.. Spokespeople.. Amy Award Winners.. When Drs.. Leslie Gordon and Scott Berns only child Sam was diagnosed with Progeria in 1998, they immediately began to collect as much information as they could find on the disease.. They discovered that there wasn t much available: there was no way to definitively test for the disease, no funding for Progeria research, and no organization advocating for children with Progeria.. So in 1999, they gathered family, friends and colleagues and established The Progeria Research Foundation to find  ...   to help advance the field of Progeria research at an extraordinary rate.. PRF s Board of Directors, Board of Advisors, Committee members, Corporate Officers, lawyers, accountants, graphic designers and public relations representatives all devote their time, energy and talents to PRF for free to ensure less is spent on administrative costs and more on raising awareness and finding a cure for Progeria.. Click on the links to the left to read more about our core group, and also read about many of our other heroes,.. PRF s Miracle Makers..

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