Author Archives: Lindsey Shapiro PhD

CANbridge Pharmaceuticals is planning to ask the U.S. Food and Drug Administration for permission to launch, by the end of 2024, clinical trials of its second-generation gene therapy candidate for spinal muscular atrophy (SMA). That request — for clearance for first-in-human studies — will come in the…

Evrysdi (risdiplam) may be associated with longer survival and greater motor improvements than Spinraza (nusinersen) in children with spinal muscular atrophy (SMA) type 1, according to an indirect comparison of clinical trial data. Sponsored by Roche — which markets Evrysdi — the analysis “leverages the longest…

Most spinal muscular atrophy (SMA) patients with four or more copies of the backup SMN2 gene achieved or maintained motor milestones after treatment with Zolgensma (onasemnogene abeparvovec), according to real-world data from the RESTORE patient registry. The one-time gene therapy from Novartis was also found safe in…

Treatment with apitegromab for two years continues to stabilize or improve measures of motor function, fatigue, and life quality in children and young adults with spinal muscular atrophy types 2 and 3 who are not able to walk. That’s according to data from the TOPAZ clinical…

Motor function is still stable or improved among people with spinal muscular atrophy (SMA) types 2 and 3 after four years of treatment with Evrysdi (risdiplam), according to updated results from the SUNFISH clinical trial. Patients and caregivers also reported continuous improvement or stabilization in the level…

The one-time gene therapy Zolgensma (onasemnogene abeparvovec) will now be routinely accessible at low or no cost to presymptomatic infants in England who are genetically predisposed to spinal muscular atrophy (SMA). Per updated recommendations from the country’s National Institute for Health and Care Excellence (NICE), this…

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…