The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

On Monday, 17 March, the Muscular Dystrophy UK President’s Awards took place, in association with Airnow Technology and ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with a ...

REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, a differentiated ...

Medication costs are only a piece of the puzzle when clinicians think about health care access, explains Leigh Maria Ramos-Platt, MD.

Ifetroban demonstrated significant 5.4% improvement in cardiac functionNASHVILLE, Tenn., March 19, 2025 /PRNewswire/ -- Cumberland Pharma ...

Hazel Green is a wide spot in the road, home to about 65 families. Like most small towns, it has seen better days. Now vacant buildings and empty lots line the main drag.

Cargo was founded by two highly acclaimed cell-therapy researchers. The company raised over $500 million to develop ...

Democrats in several states have introduced bills to strengthen protections for abortion clinics in light of recent moves by ...

Huntsville Hospital Health System (HH Health), one of the nation's largest not-for-profit health systems, is expanding its relationship with Oracle Health to help enhance patient care, accelerate ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...