Opinion: Breakthrough therapies have given Duchenne muscular dystrophy families like mine hope — and new fears
Duchenne muscular dystrophy has seen more progress in the past 15 months (give or take) than in the last couple of decades combined. In that time, the Food and Drug Administration has approved three new therapies: Elevidys (delandistrogene moxeparvovec-rokl), the first micro-dystrophin gene therapy; Duvyzat (givinostat), a non-steroidal HDAC inhibitor; and Agamree (vamorolone), a new corticosteroid.
But the currently available treatments all have limitations, many patients are not eligible, and we still have no cure. As the CEO of a biotech firm seeking to accelerate the treatment of rare diseases, and as the mother of a child with DMD, I think many in the field fail to understand that while families now have more hope, they are also more confused than ever.
