The Muscular Dystrophy Association (MDA)’s Kickstart program has announced receipt of both U.S. orphan drug and rare pediatric disease designations in support of work for congenital myasthenic ...
When the muscles needed for breathing are affected, a patient is said to be in myasthenic crisis. This is a life-threatening situation. Though anyone can get myasthenia gravis, those most likely ...
The first project within the MDA Kickstart program is focused on congenital myasthenic syndrome due to mutations in the choline acetyltransferase gene (CHAT) and likely affects less than 250 people in ...