Progeria Research Foundation

Progeria Research Foundation (PRF) is a non-profit organization dedicated to funding research for the discovery of treatments and the cure for Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS). Founded in 1999 by Dr. Leslie Gordon and Dr. Scott Berns after their son, Sam Berns, was diagnosed with Progeria, the foundation has been at the forefront of supporting and advancing research related to this rare and fatal genetic condition. Progeria is characterized by accelerated aging in children, leading to premature death usually from heart disease.

Mission and Vision

The mission of the Progeria Research Foundation is to discover treatments and a cure for Progeria and its aging-related disorders. PRF's vision encompasses not only helping those suffering from Progeria but also has broader implications for aging populations and cardiovascular diseases in the general population.

Research and Achievements

PRF has played a pivotal role in Progeria research, including the funding of the first-ever Progeria drug trial. One of the foundation's significant achievements was the discovery of the gene mutation responsible for Progeria, LMNA, which produces an abnormal form of the protein lamin A known as progerin. This discovery has opened new avenues for understanding the biology of aging.

PRF has also established the Progeria research cell and tissue bank, providing essential resources for scientists around the world. The foundation's efforts have led to the development of the first treatment for Progeria, Lonafarnib, which has shown to improve cardiovascular health and increase lifespan in children with Progeria.

Programs and Initiatives

The Progeria Research Foundation has initiated several programs to support its mission, including:

• The PRF International Registry and Medical & Research Database, which collects information from Progeria families worldwide to facilitate research and clinical trials.
• The PRF Diagnostics Testing Program, offering free genetic testing for Progeria worldwide.
• The PRF Cell & Tissue Bank, providing biological samples to researchers globally.
• Annual scientific workshops and meetings that bring together leading scientists and researchers in the field of Progeria and aging.

Fundraising and Awareness

PRF relies on donations, fundraising events, and grants to support its research and programs. The foundation organizes various events, including the annual PRF Night of Wonder gala and the Team PRF running program, to raise awareness and funds for Progeria research.

Impact on Progeria and Aging Research

The work of the Progeria Research Foundation has not only advanced the understanding and treatment of Progeria but also contributed to the broader field of aging research. Insights gained from Progeria studies have implications for understanding common diseases associated with aging, such as heart disease, stroke, and Alzheimer's disease.

Conclusion

The Progeria Research Foundation has been instrumental in bringing hope to children with Progeria and their families. Through its dedication to research, fundraising, and awareness, PRF continues to make significant strides towards finding a cure for Progeria and unraveling the mysteries of the aging process.

NIH genetic and rare disease info

• NIH info on Progeria Research Foundation

Progeria Research Foundation is a rare disease.