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…
A technique called bioelectrical impedance analysis (BIA) can be used to measure body composition in people with type 2 or 3 spinal muscular atrophy (SMA), a new study reports. The study, “A comparative analysis of body composition assessment by BIA and DXA in children with type…
Raft Digital Therapeutics has launched a demo version of Cloud Bazaar, a therapeutic video game that promotes physical activity and exercise in children with spinal muscular atrophy (SMA). Developed in partnership with Biogen Canada, the game aims to promote exercise adherence by turning physical activities into games and…
The U.S. Food and Drug Administration (FDA) has given NMD Pharma the green light to conduct a Phase 2 trial of its oral therapy NMD670 in people with spinal muscular atrophy (SMA) type 3. The upcoming clinical trial aims to evaluate the efficacy, safety, and tolerability of…
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