Spinal fusion surgery — a procedure to fuse two or more spine vertebrae — is more successful at reducing spine curvature in children with spinal muscular atrophy (SMA) type 1 and 2 if they have previously been given growth-friendly spinal implants, a study reports. But the…
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Researchers in Germany have found that newborn screening for spinal muscular atrophy (SMA) would likely be beneficial, based on evidence that early diagnosis and treatment leads to better motor development such as walking and sitting independently, less need for permanent ventilation, and lower mortality. The…
Delays in diagnosing spinal muscular atrophy are still evident, particularly for those with SMA type 3, despite recent changes in recommendations for disease detection and care, a study from Italy reports. Such delays are particularly relevant as targeted therapies are now available, and growing evidence links greatest benefit to…
The systemic nature and high specificity of risdiplam — an oral treatment under review in the U.S. to possibly treat all types of spinal muscular atrophy (SMA) — are key to its effectiveness and favorable safety profile. In an interview with SMA News Today, Paulo Fontoura, MD,…
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…
