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

For sura-vec in wet AMD, top line data from pivotal studies ATMOSPHERE and ASCENT are anticipated in Q4. First patient dosing in NAVIGATE for diabetic retinopathy is planned next quarter, expected to ...

A RECOMMENDATION for the HSE not to cover the cost of a drug to help children with a rare muscular disease has been blasted by families. Last year, The Irish Sun highlighted the plight of ...

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

When Ingram became Sarepta Therapeutics’ CEO in 2017, he didn’t have a connection to muscular dystrophy, but he has developed ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations on its RGX-202 investigational gene therapy for Duchenne muscular dystrophy at the 2026 Muscular Dystrophy Association (MDA) Clinical & ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD).

Next week, researchers, clinicians, industry leaders, and families will gather at the 2026 MDA Clinical & Scientific Conference, hosted by the Muscular Dystrophy Association (MDA), to discuss the ...

Interim clinical study results for the BB-301 Phase 1b/2a Treatment Study including 12-month follow-up results for the first four Cohort 1 ...

On March 10, 2026, from 12:00 p.m. -- 1:30 p.m. ET, in room Key West ABCD at the Hilton Orlando, Avidity will host an industry forum lunch titled "Biomarker Advancements in Rare Neuromuscular Disease: ...

A research team from London examined the relationship between adenovirus-associated virus (AAV)-based vectors and liver ...