Risdiplam continues to show promise as a therapy for individuals with spinal muscular atrophy (SMA) types 2 and 3, results from an ongoing Phase 2/3 trial show. Results from the study, “Update from SUNFISH Part 1: Safety, Tolerability and PK/PD from the Dose-Finding Study, Including…
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Risdiplam (RG7916) can improve both survival and motor function in patients with type 1 spinal muscular atrophy (SMA) beyond that expected in the natural history of the disease, according to recent clinical data. These data were presented during the 2019 annual meeting of the American Academy of…
A single administration of Zolgensma continues to extend the survival of patients with spinal muscular atrophy (SMA) type 1 while halting motor neuron loss several years after dosing, according to long-term clinical data. Zolgensma, developed by AveXis — later purchased by Novartis in 2018 — is a one-time treatment intended…
Spinal muscular atrophy (SMA) treatment with Biogen’s Spinraza (nusinersen) has resulted in motor milestone achievements in line with normal motor development and continues to provide benefit to infants who began receiving the treatment in a presymptomatic stage of the disease, according to interim clinical trial results. The study, “…
Treatment with Spinraza (nusinersen) results in greater improvements in motor function and continues to improve or stabilize motor function scores in spinal muscular atrophy (SMA) patients, according to recent clinical data. The findings were presented during the 2019 American Academy of Neurology (AAN) Annual Meeting, being…
Treatment of presymptomatic babies with the investigational gene therapy Zolgensma provides rapid improvement in motor function in infants with spinal muscular atrophy (SMA) types 1, 2 and 3, according to preliminary results of a Phase 3 trial. The study, named SPR1NT (NCT03505099), is enrolling participants…
Type 2 patients with spinal muscular atrophy (SMA) given the gene therapy Zolgensma via spinal canal injection are showing no safety concerns so far and notable motor milestones, early results of a Phase 1 trial report. Based on these interim but promising data, AveXis anticipates a request being made for the…
Long-term treatment with Biogen‘s Spinraza (nusinersen) showed clinically meaningful improvement in the motor function of children ages 2 to 15 with late-onset spinal muscular atrophy (SMA), trial data shows. Spinraza restored motor function and stabilized disease activity over long periods of time in children with type…
Learning that spinal muscle atrophy was the reason her 15-month-old son couldn’t do what others his age could was a “gut-wrenching” moment, Kristen Farrell recalled in a recent webcast hosted by Biogen. But, as Braeden turned 2, a clinical trial opened for a potential SMA treatment — known then only…
Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.
