Author Archives: Lindsey Shapiro PhD

Evaluating the strength of nerve cell impulses to muscles may be a good way of predicting motor skill recovery after Zolgensma (onasemnogene abeparvovec) treatment in infants with spinal muscular atrophy (SMA), a recent study suggests. The research showed that compound muscle action potential (CMAP) amplitudes in the…

A year of treatment with Evrysdi (risdiplam) was safe and boosted levels of SMN — the protein that’s deficient in spinal muscular atrophy (SMA) — among previously treated patients, according to published Phase 2 trial data. The safety profile was deemed similar to that observed in trials testing…

Children with spinal muscular atrophy (SMA) type 1 who start treatment with approved therapies after their symptoms emerge may be more likely to show below average cognitive, communication, and motor skills than those who start treatment before their symptoms, a study found. Cognitive function appeared to be most…

Treatment with bicalutamide and trehalose extended survival and improved motor function in a mouse model of spinal and bulbar muscular atrophy (SBMA), a study has found. Both compounds had beneficial effects on some aspects of muscle fiber structure and reduced cell death. While bicalutamide also lowered levels of…

A gene-silencing therapy designed to lower the levels of two proteins was found to improve muscle strength and coordination in a mouse model of spinal and bulbar muscular atrophy (SBMA), a new study shows. The therapy, which targets the protein lysine-specific demethylase 1 (LSD1) and the protein arginine…

People with spinal muscular atrophy (SMA) types 2 and 3 who received Evrysdi (ridisplam) in the SUNFISH clinical trial and its extension part continued to see improvements or stabilizations in motor function after two years, according to published trial data. Notably, patients who were originally assigned to…

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)…

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…