News

The U.S. Food and Drug Administration (FDA) asked for the SUNFISH clinical trial’s top-line data that necessitated a delay in its review of whether risdiplam could be an oral treatment for spinal muscular atrophy (SMA), and for good reason — these pivotal results could lead to the treatment being available…

The U.S. Food and Drug Administration (FDA) has extended its review of risdiplam as an oral treatment for spinal muscular atrophy (SMA) by three months, pushing the decision due date to August 24 from May 24. This extension was based on additional clinical data submitted by Roche — one…

Problems with the secretion of survival signals from muscle cells seems to be the dominant driver of motor neuron loss in spinal muscular atrophy lower extremity predominant 2 (SMA-LED2), an SMA subtype largely affecting the legs, a study suggests. The study, “Loss of BICD2 in muscle…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

The first patient has been dosed in the Phase 2/3 DEVOTE trial assessing the safety, tolerability, and efficacy of a higher dose of Spinraza (nusinersen) in patients of all ages with spinal muscular atrophy (SMA), Biogen has announced. Spinraza, the first disease-modifying treatment approved by…

The Muscular Dystrophy Association (MDA) is hosting an online Q&A session today to discuss the best ways that people with neuromuscular diseases might protect themselves during the COVID-19 pandemic. The 30-minute session can be accessed on the MDA’s Facebook page, and starts at 6 p.m. EST. People with…

The U.S. Food and Drug Administration has decided not to fine or otherwise sanction Novartis for manipulated data included in the application that led to Zolgensma‘s approval to treat spinal muscular atrophy (SMA) in children up to age 2. The FDA was not informed until roughly a month after it announced…

Young children given a single injection of Zolgensma into the spinal canal continue to show gains in motor function and safety, new interim data from the STRONG trial in spinal muscular atrophy (SMA) type 2 patients up to age 5 show. These findings from the open-label Phase 1/2 clinical…

The European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP), has recommended conditional approval of Zolgensma (onasemnogene abeparvovec) for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of type 1, or for those with SMA carrying up to three…

A single infusion of Zolgensma (onasemnogene abeparvovec-xioi) continues to lead to meaningful and sustained benefits in children with spinal muscular atrophy (SMA) treated in infancy, according to data from several clinical trials. Zolgensma, formerly known as AVXS-101, is a gene therapy originally developed by AveXis, now…