The team of researchers who discovered and developed Evrysdi (risdiplam) — the first at-home oral therapy for spinal muscular atrophy (SMA) — was selected as the winner of two prestigious treatment discovery awards. These are the Society for Medicines Research (SMR)’s 2021 Award for Drug Discovery and the British Pharmacological…
Scholar Rock announced the full design of the upcoming Phase 3 clinical trial testing apitegromab, its experimental muscle-directed therapy for spinal muscular atrophy, in children and young adults with types 2 and 3 SMA who are unable to walk. Apitegromab will be given as an add-on therapy to…
A CA$414,000 grant was awarded to the Maritimes by Muscular Dystrophy Canada (MDC) and Novartis Pharmaceuticals Canada to further support the implementation of newborn screening for spinal muscular atrophy (SMA). The funding (about $322,600), received by the Maritime Newborn Screening Program (MNSP), will go toward work to…
The National Institute for Health and Care Excellence (NICE) has recommended that Roche’s Evrysdi (risdiplam) be covered by England’s national health service (NHS) and provided at low or no cost to eligible spinal muscular atrophy (SMA) patients as part of a managed access agreement. The current recommendation and its…
The blood levels of neurofilaments — a marker of nerve cell damage — and the results of a nerve-muscle test called compound muscle action potential (CMAP) may serve as a biomarker of disease onset and treatment response in infants and children with spinal muscular atrophy (SMA) up to 3…
It’s strange to think that a little over two years ago, I witnessed — and participated in — the conception of SMA My Way, a patient-focused community sponsored by the biotech company Genentech to support all of those affected by SMA. A lot has changed since then. I graduated…
Motor function and stabilizing or improving breathing — along with oral administration — are the factors that adult patients and caregivers of children with spinal muscular atrophy (SMA) type 2 or 3 who cannot walk value most when deciding on available treatments, a U.K. survey study shows. Avoiding…
Roche and its subsidiary Genentech are launching a Phase 2/3 clinical trial to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody, in combination with Evrysdi (risdiplam) in children with spinal muscular atrophy (SMA). The MANATEE trial has two parts. In part one, 36…
A pilot newborn screening (NBS) program for spinal muscular atrophy (SMA) that started in Liège province has transitioned into an official, government-run program spanning Southern Belgium. Its architects described their experience, highlighting concepts that may be useful for creating similar programs, as well as problems that arose and how they…
A branch of the Canadian Agency for Drugs and Technologies in Health (CADTH) is recommending that Evrysdi (risdiplam) be added to public health plans across Canada, making it available to spinal muscular atrophy (SMA) patients at low or no cost between 2 months and 25 years of age, provided…