Columnist Betty Vertin has begun thinking about what she'd want in a new home to better accommodate three young men with Duchenne MD.

Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living ...

PPMD is proud to share our partnership with the Cooperative International Neuromuscular Research Group (CINRG) to advance the expanded Duchenne Natural History Study (eDNHS) study. Joining PPMD in ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and ...

Q4 2025 . Management View. Curran Simpson, President and CEO, stated, "2026 is set to be a pivotal year for REGENXBIO with great focu ...

The agency acted after reviewing mouse data submitted in 2024, making the timing of the new pause “confusing,” one analyst wrote.

An executive assistant's rapid rise and unexpected departure fueled a seismic shift in Michigan football, triggering ...

SonoThera ™, a biotechnology company dedicated to addressing the root causes of disease through next-generation genetic medicines, today announced it will present at the annual Muscular Dystrophy ...

Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA) New Phase 1b data further illustrate potential of salanersen in SMA, ...

The FDA has put a partial clinical hold on PepGen’s phase 2 mus | The FDA has put a partial clinical hold on PepGen’s phase 2 muscle wasting trial after reviewing preclinical data, continuing the stop ...

Sarepta has come under scrutiny over Elevidys, its gene therapy for Duchenne Muscular Dystrophy (DMD). With a one-time ...

Corpus Christi firefighters launch the 2026 "Fill the Boot" campaign to support MDA families in the Coastal Bend on three ...