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

Biogen has launched a trio of Phase 3 trials to test salanersen, a new SMA treatment that aims to be a more convenient ...

Jesy Nelson broke down in tears after her campaign for more health checks on babies reached 100,000 signatures - meaning it ...

Union Minister H D Kumaraswamy has assured assistance for a two-year-old child suffering from a rare genetic disorder after ...

NMD Pharma will also present an update on the ongoing SYNAPSE-MG Phase 2b clinical trial of ignaseclant in generalized myasthenia gravisAarhus, ...

In Trumbull County, a local nonprofit will once again be hiding colorful candy-filled eggs in yards this Easter to help a ...

Easter Egg fundraiser to benefit 9-year-old Trumbull County girl with Spinal Muscular Atrophy ...

Little Mix singer Leigh-Anne Pinnock has opened up on her friendship with former bandmate Jesy Nelson and her twins' devastating diagnosis ...

The singer, 34, said she had reached out to her former bandmate after she found out her twin daughters may not live beyond the age of two after being diagnosed with genetic neuromuscular disease, ...

The singer, 34, said she had reached out to her former bandmate after she found out her twin daughters may not live beyond the age of two after being diagnosed with genetic neuromuscular disease, ...

Gemma Biotherapeutics ("GEMMABio"), a clinical‑stage global genetic medicines company, announced today that the first patient has been dosed in the Phase 1/2 CHARISMA clinical trial of GB221, an ...