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…
Spinraza (nusinersen) may restore the normal development of motor neurons — the nerve cells responsible for making muscle cells work — in children with spinal muscular atrophy (SMA), particularly those who start treatment early, a study found. Besides getting motor neurons back onto their developmental trajectory, Spinraza also may slow their…
A song written and performed by an artist with spinal muscular atrophy (SMA) is the latest project to come out of Genentech’s SMA My Way program, a collaboration that allows those in the SMA community to share knowledge and experiences with others affected by the disease, as well…
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…
One of NBC’s newest TV shows, “Ordinary Joe,” is making a mark in the spinal muscular atrophy (SMA) community for including a character with the disease. The drama series follows protagonist Joe Kimbreau, played by James Wolk, through three separate realities stemming from a single decision he made…
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…
Spinraza (nusinersen) improved fine manual dexterity — the ability to make coordinated finger movements to grasp and manipulate objects — in both hands of five children with spinal muscular atrophy (SMA) type 2 over 1.5 years of treatment, a case series study showed. This study “is the first…
Delayed Spinraza (nusinersen) treatment due to the COVID-19 pandemic did not directly result in a worsening of symptoms in children with spinal muscular atrophy (SMA), a small study suggests. Any worse motor function that occurred during this period was attributed to a lack of response to the therapy…
Genetic analysis of dried saliva spots identified people with spinal muscular atrophy (SMA), a study demonstrated and possibly for the first time. The scientists noted that this noninvasive and low-cost, alternate method of genetic screening may be suitable for programs held in schools or workplaces without the need for…
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