Owned Asset Benefits From Existing Ophthalmology and Manufacturing ExpertiseFuchs Endothelial Corneal Dystrophy (FECD) ...

Lineage’s COR1 targets restoration of endothelial pump function via intracameral delivery and adhesion to Descemet membrane, ...

Sarepta Therapeutics’ next crop of medicines for muscular dystrophy appear to be safe and effective in early clinical trials, ...

Shares of Dyne Therapeutics gained after the company shared positive data from a continuing trial of a potential Duchenne muscular dystrophy treatment. The stock rose 14%, to $16.93, midday Monday.

Agency officials promise fast reviews of new treatments while vowing they will not be a “rubber stamp” for the industry. But patients with rare diseases view recent decisions as signs that the doors ...

- New analyses out to 24-months showed improvement in heart and lung function compared to expected declines in DMD natural history - - Data expand on previously reported results demonstrating that ...

PARAMUS, N.J., March 9, 2026 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma, New Jersey, USA; President, Yukiteru Sugiyama ), a biopharmaceutical leader in rare diseases ...

Several abstracts, including a late-breaking podium presentation and posters, bring forward accumulating long-term efficacy, safety and caregiver-reported insights that deepen understanding of ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking ...

Pratteln, Switzerland, February 27, 2026 – Santhera Pharmaceuticals (SIX: SANN) today announces that the Spanish Interministerial Commission on Prices of Medicines, has proposed to include AGAMREE® ...

When David Fajgenbaum and his colleagues launched the drug repurposing non-profit Every Cure, they chose a path that ...

New treatments for neurodegenerative diseases like Alzheimer's, Parkinson's, and motor neuron disease (MND) could be unlocked ...