When I was diagnosed with ALS, I was hoping to see my sons graduate from high school. As it turns out, I’ve been blessed to have seen them graduate from high school and college.
I know I’m one of the lucky ones. ALS has progressed fairly slowly in me and I don’t take that for granted. From day one of learning I have this disease, I’ve done what I can to advance ALS research and the discovery of new treatments.
All of us living with ALS have to have hope: hope for a treatment and hope for a cure. That’s why I’m excited about a new report that came out today from the Pharmaceutical Research and Manufacturers of America (PhRMA) and The ALS Association, “Medicines in Development for Rare Diseases.”
This new report, which also shares my story, finds that America’s biopharmaceutical research companies are currently developing more than 560 medicines for patients with rare diseases, 38 of which are for people with neurological disorders, including ALS.
Among the new therapies in development for ALS is antisense technology against SOD1. Antisense technology is an important step toward helping patients and their families manage ALS and something The ALS Association has supported since 2003.
There are about 7,000 known rare diseases that affect fewer than 200,000 people in the U.S. every year. This report gives me hope that industry is focusing on these diseases, including ALS, more than ever before.