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…
News
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…
Various ethical and social factors influence care decisions for families affected by spinal muscular atrophy (SMA) in this new era of disease-modifying therapies (DMTs), according to a Canadian study. In interviews, caregivers reported that a range of factors, including financial resources and their expectations of treatment outcomes, influenced their…
Biogen has acquired Alcyone Therapeutics and will continue to develop ThecaFlex DRx, a device intended to ease patient experience and accessibility when delivering certain medicines into the spinal canal. The ThecaFlex DRx System is initially being evaluated with Spinraza (nusinersen) in people with spinal muscular atrophy (SMA),…
Measuring the levels of the survival motor neuron (SMN) protein in blood cells may help track spinal muscular atrophy (SMA) patients’ response to Evrysdi (risdiplam), a study from Japan found. Patients taking the drug showed a significant increase in blood levels of SMN after one month of therapy. SMN…
An experimental oral combination of two therapies eased motor symptoms and prolonged survival in a mouse model of spinal muscular atrophy (SMA), a study shows. The study, “The effect of coadministration of D156844 and AR42 (REC-2282) on the survival and motor phenotype of mice with spinal muscular atrophy,”…
