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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…
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)…
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…
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…
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…
As previously mentioned in one of my recent articles entitled “Post-Surgery Care for SMA Patients Taking Physical Therapy,” Michael, my patient with SMA, recently went through an elective surgery to have a subcutaneous port implanted in his chest, to reduce difficulty receiving medications through intravenous lines. With this surgery,…
Severe scoliosis — an abnormal curvature of the spine — was significantly associated with older age and limited motor abilities in children with spinal muscular atrophy (SMA) type 2 who had not received disease-modifying therapies, a study showed. These findings establish characteristics of untreated scoliosis progression on SMA…
SMA News Today · #118: Aging and SMA Long before Scholar Rock began its Phase 3 pivotal trial of apitegromab, the FDA had awarded Scholar Rock with…
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