First patient dosed in ELEVATE-44-201 with data from first patient cohort anticipated in H1 2026 -- -- Initiated ELEVATE-45-201 and on track to dose the first patient in Q3 2025 -- -- Multiple ...

Cumberland Pharmaceuticals' Q2 2025 earnings highlight revenue growth, clinical progress in ifetroban for DMD, and Vibativ's global expansion.

The FDA is allowing Sarepta to resume shipments of Elevidys (delandistrogene moxeparvovec) to ambulatory patients with Duchenne muscular dystrophy.

A 12-year-old boy from Carthage, Tennessee finds joy in his power wheelchair. His family are hoping for a cure for his muscular disease, Duchenne.

Deklan Locke, whose nickname is Flash, has a rare illness, Duchenne muscular dystrophy, which causes progressive muscle ...

A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular ...

Precision BioSciences Inc., a clinical-stage company developing gene editing therapies for diseases with high unmet need, has ...

Dyne Therapeutics' DYNE-251 receives FDA breakthrough status for Duchenne muscular dystrophy treatment. Read more here.

Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the ...

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...

Targeted rewriting of the epigenome promises to overcome several challenges in direct gene editing in patient therapies.