When you're diagnosing a child with Duchenne muscular dystrophy, says Alexandra Bonner, MD, of Cleveland Clinic, it's important to not only determine if she's a carrier of the gene mutation that ...

The first gene therapy that can treat Duchenne muscular dystrophy (DMD) has been approved by the U.S. Food and Drug Administration; it will be marketed as Elevidys (delandistrogene moxeparvovec-rokl) ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the ...

A new gene therapy treatment gained FDA approval last month to treat boys ages four and five with Duchenne muscular dystrophy, a rare and deadly genetic disorder that leads to muscle degeneration.

Dyne Therapeutics started the new year with proof-of-concept clinical data for experimental genetic medicines addressing two rare muscle diseases—one of which has no FDA-approved treatments. Now the ...

The FDA approved the first gene therapy for some pediatric patients with Duchenne muscular dystrophy, a rare genetic condition that weakens the body’s muscles, on June 22. The approval is relatively ...

Interim data showed 110% average microdystrophin expression and up to 88% dystrophin-positive muscle fibers in the first three participants. Two patients saw an 8% average heart function improvement ...

Solid Biosciences ((SLDB)) announced an update on their ongoing clinical study. Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst ...

Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic ...

Genethon is uniquely positioned to deliver long-term value and strategic collaboration opportunities in gene therapy.

—Starting cardiac medications, using the proper timing and dosing, to optimize the efficacy of therapies and improve patient outcomes, is key to cardiac care for patients with Duchenne muscular ...