Investigational RGX-202 continues to demonstrate evidence of positively changing disease trajectory for Duchenne Pivotal dose participants exceeded external controls across functional ...

SGT-003 in the INSPIRE DUCHENNE trial to date - - Robust microdystrophin expression with consistent mechanistic evidence of dystrophin associated protein complex (DAPC) restoration and improvements in ...

Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases.

People with BMD who received sevasemten in clinical trials maintained stable motor function over several years of follow-up, new data show.

Experts highlight the role of primary care in identifying joint hypermobility syndrome and distinguishing it from connective tissue disorders.

Incubated by Atlas Venture, the biotech startup is advancing a daily pill it sees potentially helping people with dystonia or ...

The following segment was excerpted from the Harbor Health Care ETF Q4 2025 Commentary.During the fourth quarter, the Harbor Health Care ETF ...

Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and Vinay Prasad, director of the Center for ...

The MESA data show stabilization of function in participants on sevasemten versus the predicted functional decline observed in Becker natural history ...

Sildenafil—an active ingredient also marketed under the name of Viagra—improves symptoms in patients with Leigh syndrome. This has now been reported in the journal Cell by researchers at ...

- Company to highlight new safety, pharmacokinetic, and pharmacodynamic data from Phase 1 clinical trial of ARV-102 in participants with Parkinson’s disease - NEW HAVEN, Conn., March 11, 2026 (GLOBE ...

Researchers are to test whether statins could help people with motor neurone disease (MND) live longer after data suggests cheap cholesterol-busting drug may 'prolong survival'. MND is a progressive ...