Patients with Duchenne muscular dystrophy (DMD) are prescribed corticosteroid anti-inflammatory drugs as standard of care, but long-term use comes with significant adverse effects. For individuals ...

BOULDER, Colo., March 10, 2026 /PRNewswire/ -- Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today presented at the 2026 MDA Clinical and Scientific ...

Dyne Therapeutics reports positive long-term data for DYNE-251 in Duchenne muscular dystrophy, aiming for U.S. accelerated approval in 2026. Dyne Therapeutics announced new long-term clinical data ...

SAN DIEGO, March 17, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare ...

SAN DIEGO, Oct. 11, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (CAPR) (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of ...

RegenXBio Inc. ((RGNX)) 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 insights ...

SAN FRANCISCO -- Givinostat (Duvyzat) had a manageable long-term safety profile in Duchenne muscular dystrophy, data from an open-label extension study suggested. Most treatment-emergent adverse ...

Comparable long-term effectiveness to classic corticosteroids, both prednisone (p=0.8587) and deflazacort (p=0.6544), based on time to loss of ambulation 80% fewer patients with vertebral fractures (8 ...

Please see Important Safety Information below and the full Prescribing Information. CONCORD, Mass., Aug. 25, 2025 /PRNewswire/ -- ITF Therapeutics LLC, the U.S. affiliate of Italfarmaco, today ...

REGENXBIO Inc. RGNX on Thursday released topline and interim functional data from the pivotal Phase 1/2/3 AFFINITY DUCHENNE ...

-Results in Performance of the Upper Limb (PUL v2.0) Continue to Show Slowing of Disease Progression in Later-Stage DMD Patients- -Improvements Seen in Multiple Cardiac Endpoints Demonstrating ...

LV end systolic volume, indexed (mL/m 2 )-2.4-5.1 LV end diastolic volume, indexed (mL/m 2)-5.7-10.0 Click to enlarge *Baseline is referred to as start of HOPE-2 study, changes in medians are shown; 3 ...