Jensen Caraballo, a beloved disability rights advocate in New York, has died at age 33. Born with spinal muscular atrophy, ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

Parents of children with spinal muscular atrophy (SMA), a rare disease, welcomed the announcement by the Indian pharma ...

Scholar Rock Holding Corporation's apitegromab shows promise for SMA treatment, aims for 2025 launch. Click for my updated ...

The state govt provides free treatment to numerous children with rare diseases like Spinal Muscular Atrophy (SMA), growth ...

Thiruvananthapuram: Health minister Veena George announced the launch of the Vishu Kaineettam project, aimed at supporting ...

The issues at Temple Street in a HIQA report published this week relate to a consultant, identified as Surgeon A, and ...

PTC Therapeutics, Inc.’s PTCT share price has surged by 6.72%, which has investors questioning if this is right time to sell.

SMA has rendered me unable to perform most everyday, ordinary tasks unassisted. Consequently, I can’t be left alone. To be ...

Learn more about whether Neurocrine Biosciences, Inc. or PTC Therapeutics, Inc. is a better investment based on AAII's A+ ...

Evidence suggests inbreeding heightens the risk of birth defects and developmental delays, such as learning and speech ...