'My life is complete.’ ...

Spinal muscular atrophy (SMA) is a rare, genetic condition that, across all types, affects ~1,600 people in the UK.1 It can cause muscle weakness which gets worse over time, causing movement, ...

Children with spinal muscular atrophy (SMA), a rare autosomal recessive disease that results in progressive muscle weakness and atrophy, can suffer the concerning consequences of dysphagia, which ...

A multidisciplinary team at Medcare delivered the therapy, led by its Gene Therapy Unit clinical lead, Dr. Vivek Mundada, ...

University of Pittsburgh School of Medicine researchers carrying out a small pilot clinical trial demonstrating that a drug-free, minimally invasive intervention targets the root cause of progressive ...

Cenrifki prevents MS disease progression and Itvisma delivers a healthy gene-to-cell nuclei, addressing the root cause of SMA.

- Taldefgrobep alfa, a myostatin-targeting biologic investigational agent, in Phase 3 development to increase muscle mass for Spinal Muscular Atrophy patients now granted Fast Track in addition to ...

If approved, Itvisma (intrathecal onasemnogene?abeparvovec) will be first and only gene replacement therapy for children two years and older, teens and adults with SMA in the European UnionItvisma dem ...

This marks a significant advancement in gene therapy, as this patient is the second adult SMA patient worldwide to be treated ...