Major UK study to evaluate newborn screening for spinal muscular atrophy

SENS study to assess feasibility, effectiveness and family experience of introducing SMA screening into the NHS newborn screening programme

Servier to acquire Edgewise muscular dystrophy business in $2.65 billion deal

The transaction includes an upfront payment of $1.55 billion, along with up to $1.1 billion in regulatory and commercial milestone payments

Biogen bags FDA nod for high dose SPINRAZA in spinal muscular atrophy

The new regimen, delivering 50 mg/5 mL and 28 mg/5 mL doses, offers a higher concentration of the drug for both loading and maintenance phases

Capricor's cell therapy shows breakthrough results in Duchenne muscular dystrophy trial

It's a development that marks a potential breakthrough for people living with the rare and debilitating disease Duchenne muscular dystrophy (DMD)

Merck KGaA’s Cladribine capsules gain FDA fast track for rare neuromuscular disorder

Delhi High Court refuses to restrain Natco from manufacturing spinal muscular atrophy drug

The Court dismissed Swiss drugmaker Roche’s plea for interim injunction

FDA grants ODD to Keros KER-065 for treatment of Duchenne muscular dystrophy

KER-065 is a novel ligand trap comprised of a modified ligand-binding domain derived from activin receptor type IIA and activin receptor type IIB

Roche updates on Elevidys gene therapy for Duchenne muscular dystrophy in EU

Elevidys is currently the only approved gene therapy that targets the root cause of Duchenne

Capricor receives FDA’s CRL for Deramiocel BLA for duchenne muscular dystrophy

Capricor’s BLA for Deramiocel received Priority Review in March 2025

Roche’s Evrysdi tablet approved by European Commission as first and only for Spinal Muscular Atrophy

Simplified storage and administration of new tablet formulation may provide greater freedom and independence for people with Spinal Muscular Atrophy (SMA)