Treatment with the muscle strengthener pyridostigmine may reduce patient-reported fatigue among people with spinal muscular atrophy (SMA), results from a small clinical trial suggest. Still, the treatment failed to ease motor fatigue and improve motor function compared with a placebo — which were the main goals of the…
In an agreement meant to provide Ionis Pharmaceuticals with additional money to advance potential therapies for various diseases, the company is giving Royalty Pharma a percentage of the payments it receives from sales of Spinraza (nusinersen), an approved therapy for spinal muscular atrophy (SMA). In exchange, …
The ongoing SAPPHIRE clinical trial testing apitegromab, Scholar Rock’s investigational muscle-directed therapy, in people with spinal muscular atrophy (SMA) is expected to complete patient enrollment later this year, the company announced. Top-line data from the trial are anticipated in 2024. Should results be positive, Scholar Rock expects…
CANbridge Pharmaceuticals has acquired exclusive global rights to develop, manufacture, and commercialize a second-generation gene therapy for spinal muscular atrophy (SMA). The company had completed preclinical studies of the investigational gene therapy in collaboration with the Horae Gene Therapy Center at the University of Massachusetts (UMass) Chan…
BraunAbility has partnered with the nonprofit organization Laughing at My Nightmare (LAMN) to donate a wheelchair-accessible vehicle to a 6-year-old girl with spinal muscular atrophy (SMA). Fynlee Armstrong, a “sassy and spunky little girl” from Clyde, Texas, requires a wheelchair to move around. Over the last few years,…
Biogen is collaborating with Alcyone Therapeutics to develop an implantable medical device that more easily delivers antisense oligonucleotide (ASO) therapies to the spinal canals of people with certain neurological disorders. Under the terms of the agreement, Biogen will acquire exclusive global rights to Alcyone’s ThecaFlex DRx system in…
A significant proportion of people with spinal muscular atrophy (SMA) do not remain on Spinraza (nusinersen) or receive treatment injections as prescribed, according to a U.S. insurance claims analysis. “SMA can be treated, but it is important that patients receive their scheduled doses of medicine as prescribed and…
The team at SMA News Today brought you daily coverage of the latest developments in treatment and advancements in research related to spinal muscular atrophy (SMA) in 2022. We look forward to continuing to serve the SMA community in the new year. Here we’ve compiled a list of…
A man in Brazil was found to have an exceptionally rare form of spinal muscular atrophy (SMA) — called SMA with lower extremity predominance, or SMA-LED — caused by a mutation in the TBK1 gene, as described in a new case report. Mutations in the TBK1 gene have…
Children with spinal muscular atrophy (SMA) type 2 and 3 have lower health-related quality of life (HRQoL) compared with healthy youngsters, but scores are similar for both patient groups, a new study reported. While disease type was not found to impact HrQoL, some health-related quality of life measures in…
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