End the Legacy, Dedicated to Providing Resources to and Advocating for the Genetic ALS and FTD Community, Presenting to FDA January 12
Summary
www.eventbrite.com/e/502784390647 During the planned presentation, community members impacted by mutations in C9orf72, GRN, Sod1, and Tardpb will discuss their experiences and perspectives losing multiple family members to these diseases and being at risk for them especially related to trial participation and early treatment with approved medications.Dr Heiman-Patterson and Jean became connected serving as joint co-chairs of an NIH working group of the National Institutes of Health ALS Strategic Plan. To this end she will be organizing the medical community to discuss guidance in the care of pre-symptomatic gene carriers and has lent support to the efforts of End the Legacy through the ALS Hope Foundation, an organization co-founded by Dr. Heiman-Patterson and dedicated to make a difference to people living with ALS.“The ALS Hope Foundation is proud to expand our programs through End the Legacy to include education and advocacy for the familial ALS and FTD community and especially those people who carry genetic changes that put them at high risk of developing the disease. The genes responsible for this inheritance became known in the 1990s, and are identified in most ALS and FTD cases where the patient has a clear family history of the disease and in small but significant numbers of ALS and FTD patients who do not report a family history. Most of the genes are autosomal dominant meaning children of patients with the mutation have a 50/50 chance of inheriting it. The importance of efforts at education, support and care for presymptomatic gene carriers is underscored by the calculated hundreds of thousands of people in the US that are affected.
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