For School Reports
“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.
Did you know?
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. 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 WILL find the cure.
1. 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 The Connection to Other Diseases , and The Science Behind Progeria. The information on those pages is not repeated here.
If you are looking for more advanced, scientific information, please visit https://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 New in Progeria Research section.
Also, you can access information on the NORD (National Organization of Rare Diseases) web site. Go to https://www.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.
Dr. Leslie Gordon, PRF’s Medical Director, wrote a chapter on Progeria for the World Book Online Reference Center World Book Progeria and the print edition of the 2008 World Book Encyclopedia
Dr. 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.
2. I heard that the Progeria gene had been discovered, where can I find out more about this?
On April 16, 2003, it was announced that the gene for Progeria had been discovered, and PRF played a major role in that discovery! Go to Progeria Gene Discovered for more information. Also, here are some cites to scientific articles on the gene mutation:
“Recurrent de novo point mutations in lamin A cause Hutchinson-Gilford Progeria Syndrome”, Vol. 423, May 15, 2003, Nature.
“Mutation causes early-aging syndrome”, Vol. 163, p.260, April 26, 2003, Science News.
“Cause of Progeria’s Premature Aging Found; expected to Provide Insight into Normal Aging Process”, Vol. 289, no. 19, pp. 2481-2482, May 21, 2003, JAMA.
3. Is Progeria a dominant or recessive disease?
It is Dominant.
4. I need to interview a researcher or other expert in the field of Progeria.
Unfortunately, due to the large number of requests and our small staff, we cannot provide interviews and we do not provide the contact information of others. However, our Medical Director Dr. Leslie Gordon has helped create this Q and A and all other information on Progeria that appears on this site, so we hope that is sufficient to satisfy the interview requirement for your report. Dr. Gordon is a leading expert on Progeria; she is the director of all of PRF’s research-related programs including our International Progeria Registry and Diagnostics Testing program, has authored dozens of scientific publications that appear in peer-reviewed, well-respected journals, has been one of the leaders in all of the Boston clinical drug trials, and has examined more children with Progeria than anyone in the world.
In addition, click here to view a 1-hour presentation by PRF co-founders Dr. Leslie Gordon (PRF’s Medical Director) and Dr. Scott Berns (PRF’s Chairman of the Board)
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?
9. Does the disease affect their mental abilities?
Not at all. Children with Progeria are intelligent and full of energy just like other kids their age.
10. Why do children with Progeria age in body so rapidly and not in mind?
LMNA is not expressed by the brain cells, so the gene mutation does not affect the brain.
11. What issues do you think are most important in the care of a patient with Progeria?
Most of all, it is important to remember that all children with Progeria have age appropriate intellect and personality. An eight-year-old with Progeria will think and act just like every other eight-year-old. Children with Progeria are smart and funny and full of life. It’s the bodies of these children that are laden with the genetic predisposition toward conditions of aging and heart disease, not their minds. So they need their schools and communities and their friends to treat them just like any other child (with a few minor adjustments for size). After all, Progeria is just a small part of who they are!
12. Are there any treatments available?
Yes. In November, 2020, PRF achieved an important piece of our mission: the first-ever treatment for Progeria, Zokinvy (lonafarnib) was granted approval by the United States Food & Drug Administration (US FDA). With this achievement, Progeria now joins less than 5% of the rare diseases with an FDA-approved treatment.*
In 2019, The Progeria Research Foundation published our second edition of The Progeria Handbook, for families, physicians, schoolteachers and others caring for children and young adults with Progeria. With 33% more material, this 131-page updated version illustrates how far we have come in our clinical understanding of Progeria since the first edition was published in 2010. Additions include sections on genetics and genetic counseling, the lonafarnib treatment, and new cardiovascular recommendations for caregivers.
We don’t have a cure yet, but we do know that daily care makes a huge difference in their quality of life. Proper nutritional, physical, and occupational therapies, along with preventative cardiac and other care are essential. The handbook is available in English, Japanese and Spanish.
*300 rare diseases that have an FDA-approved treatment (https://www.rarediseases.info.nih.gov/diseases/FDS-orphan-drugs)/7,000 rare diseases for which the molecular basis is known (www.OMIM.org) =4.2%
13. Are any scientists close to finding a 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.
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.
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.
16. I saw HBO Film’s Life According to Sam and/or a program on TV and would like a copy of the tape.
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.
17. 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).
18. 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.
19. I’d like to be a pen pal to a child with Progeria or send them a present.
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 Meet the Kids page 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.
20. I would like to talk to someone by phone or in person about living with Progeria.
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.
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?
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. Children, please ask your parents for their permission and assistance before volunteering.
22. What can people do to help? Where do you get most of your funding?
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.
23. Is there a chapter near where I live that I can contact?
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 WILL find the cure!
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.