SMA mutations disrupt spinal cord development, with fewer nerve cells in early stages. SMN protein deficiency leads to a mesodermal fate bias, with fewer motor neurons. Increasing SOX2 pathway ...

Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies for spinal muscular atrophy, Duchenne muscular dystrophy and Becker muscular ...

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, ...

Salanersen, a treatment candidate for spinal muscular atrophy (SMA) now in clinical testing, is safe and was shown to stabilize or improve motor function over at least one year of follow-up in ...

MDA 2026 keynote speaker John Crowley discussed with MD News Today the progress made in neuromuscular disease and the future ...

The president and CEO of the Muscular Dystrophy Association talks about what she's most excited about in neuromuscular disease research.

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Biogen’s salanersen is now heading into three Phase III trials in SMA.

Insmed has launched a first-in-human Phase 1 trial testing its experimental ALS gene therapy INS1202 in people with the disease.

New Phase 1b data support the safety and effectiveness of salanersen over one year in children with SMA who had the potential for improvement due to suboptimal clinical status with prior gene ...

US biotech major Biogen has presented additional results from the Phase Ib study of salanersen, an investigational novel antisense oligonucleotide (ASO) given once a year for spinal muscular atrophy ...

Investigation Discovery’s The Curious Case Of… The Woman Dying for Attention examines how Sarah Delashmit faked cancer and other illnesses for years, faced federal fraud charges, and what her life ...