A preclinical study found that blocking the SMPD1 enzyme with available drugs such as the antidepressant clomipramine increases the levels of SMN, the protein missing in spinal muscular atrophy (SMA), potentially offering a new treatment strategy. “The strategy presented here represents not only a new therapeutic approach for this…
SMA Europe has launched an online tool designed to help patients, doctors, and others in the spinal muscular atrophy (SMA) community more easily explore ongoing clinical trials across Europe and worldwide. The organization said it aims for the Clinical Trials Finder to become “the go-to global resource”…
The “Tales from the Magic Keep” exergame — a video game that uses body movements to promote physical activity — proved to be a feasible and engaging home-based tool for children and adolescents with spinal muscular atrophy (SMA), according to a small Canadian study. However, improving “gesture recognition is imperative…
Using a specially designed back brace in addition to taking part in physical therapy may work best to reduce abnormal spine curvature, or scoliosis, in children with spinal muscular atrophy (SMA) type 1, a new study from Turkey reports. During eight weeks of daily physical therapy, which…
ARD-1676, a medication that works by tagging toxic proteins for destruction in cells, promotes clearance of the androgen receptor (AR) protein, which is faulty and clumps in spinal and bulbar muscular atrophy (SBMA), a study found. Earlier approaches to finding a treatment for SBMA have had little success in…
A technique to estimate the health of nerve cells that control movement may help predict how strongly some individuals will respond to the spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi), a study reports. This metric, called the cumulative motor index (CMI), assesses four major movement-related nerves.
For adults with spinal muscular atrophy (SMA), a genetic disease that progressively weakens breathing muscles, the approved treatment Spinraza (nusinersen) offers a crucial long-term benefit — stable lung function over several years. Data from a multi-center observational study also found that a patient’s initial breathing capacity is the…
The U.S. Food and Drug Administration (FDA) has declined to approve a higher dose of nusinersen — the medication in Biogen’s long-approved therapy Spinraza — for the treatment of spinal muscular atrophy (SMA). According to Biogen, no issues were raised with the clinical data in the company’s submission seeking regulatory…
Brian Lin is watching how science is improving spinal muscular atrophy (SMA) treatment today and making advancements with the potential to reshape SMA care for tomorrow. “The most significant breakthroughs actually happened fairly recently, over the last five years,” says Lin, the research portfolio director at the Muscular…
The U.S. Food and Drug Administration (FDA) has declined to approve apitegromab as a treatment for spinal muscular atrophy (SMA). The FDA said it could not approve developer Scholar Rock‘s application at this time because of observations identified during a routine general site inspection of Catalent…
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