News

PROTAC therapy shows promise for SBMA, study finds

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…

Metric may predict response to SMA gene therapy Zolgensma

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.

Spinraza can protect adults with SMA from breathing decline

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…

FDA denies approval for higher dose of nusinersen for SMA

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…

Hope on the horizon: Science’s promise for spinal muscular atrophy

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…

SMA caregivers face complex decisions in new era of treatment

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 acquires Alcyone, device aimed at improving drug delivery

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),…

Blood test may help track response to SMA treatment Evrysdi

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…