The National Ataxia Foundation (NAF) recently collaborated with members of Congress to conduct an informational meeting about Spinocerebellar Ataxia (SCA). SCA encompasses a group of rare, debilitating, and hereditary neurodegenerative diseases. These diseases often affect multiple family members concurrently, and currently, no known cures or FDA-approved treatments exist. The meeting aimed to educate legislators, staff, and other participants about the disease and highlight the urgent need for research and treatment options.
The meeting emphasized the difficulties rare diseases like SCA encounter, particularly in clinical trials. This challenge necessitates flexibility from the FDA when evaluating new treatment applications. A primary objective of the meeting was to underscore the critical role of research in discovering effective treatments.
Bill Nye, known for his science advocacy, attended the meeting to share his family’s multi-generational experience with a recently identified form of SCA, designated SCA 27B. This specific ataxia type impacts walking, fine motor skills, and overall coordination. While the recent identification of the genetic sequence responsible for their ataxia represents a significant advancement, it’s only the initial step toward understanding and managing the condition.
Lauren Sormani provided a personal account of living with SCA, highlighting its devastating effects on individuals under 30. Lauren Moore, PhD, Chief Scientific Officer of NAF, discussed the generational impact of SCA on her own family, further emphasizing the significant need for treatments.
The NAF remains dedicated to engaging with Congress and its staff to raise awareness about SCA patients’ challenges. They advocate for increased research funding and the development of treatment options. The organization is committed to accelerating treatment development and ultimately finding a cure for ataxia.
Ataxia is a rare neurological condition affecting thousands in the U.S. and globally. This progressive disease impairs walking, talking, and fine motor skills. Established in 1957, the NAF is a non-profit organization striving to improve the lives of those affected by ataxia. The Foundation pursues its mission through funding research, providing support services for affected families, and collaborating with pharmaceutical companies in the search for treatments and a cure. The NAF actively collaborates with leading ataxia researchers worldwide, promoting the exchange of ideas and fostering innovation in ataxia research.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
