Johnny Quintana’s life changed at the age of 19 when he was diagnosed with a rare form of muscular dystrophy called Facioscapulohumeral muscular dystrophy (FSHD).
A Minnesota mother and son urge action as National Institute of Health restrictions disrupt research vital to Muscular ...
The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...
The data showed reduced difficulties in standing, walking and running that were statistically significant, the company said.