Author Archives: Marisa Wexler, MS

A medicine called leuprorelin acetate, approved in the U.S. to treat prostate cancer, can slow the progression of physical disability in people with spinal and bulbar muscular atrophy (SBMA), according to an analysis of a real-world study…

Last month’s approval of Novartis‘ one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) for people with spinal muscular atrophy (SMA) ages 2 and older is a milestone that “ushers in a new era of informed…

After a “constructive and collaborative in-person” meeting with the U.S. Food and Drug Administration (FDA), Scholar Rock is now planning to once again seek approval of its muscle-strengthening agent apitegromab as a treatment for spinal muscular atrophy (SMA). That resubmission to the FDA is expected in…

The experimental therapy BIO101 may help improve muscle strength in people with spinal muscular atrophy (SMA), according to new research done in laboratory models. “We demonstrated that a daily oral administration of BIO101 can specifically improve…

Treatment with Spinraza (nusinersen) may help stabilize lung function in people with spinal muscular atrophy (SMA) types 2 and 3, a study suggested. “Treatment of SMA types 2 and 3 with nusinersen may stabilize respiratory…

Mutations that cause spinal muscular atrophy (SMA) may trigger problems with how bone cells communicate, leading to abnormally weakened bones, according to a study done in mice and cells. The findings suggest that treatments aiming to…

People with spinal muscular atrophy (SMA) may show subtle abnormalities in eye movements, but these abnormalities are unlikely to be caused by weak muscles; instead, they likely arise due to changes in the brain, a study found.

Nearly all children with spinal muscular atrophy (SMA) who were started early on Evrysdi (risdiplam) in a clinical trial — as newborns, before the onset of symptoms — are now walking after three…

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…

Scholar Rock plans to have the the muscle-strengthening therapy apitegromab available commercially in the U.S. before the end of the year if the U.S. Food and Drug Administration (FDA) approves it next month…