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With the U.S. Food and Drug Administration (FDA) expected to soon decide whether Zolgensma (onasemnogene abeparvovec) can treat people with spinal muscular atrophy (SMA) type 1, clinical trials of the gene therapy are showing rapid benefits across disease types. Those gains might justify a broader label — one allowing…

Patients of all ages with spinal muscular atrophy (SMA) will now be able to be treated with Spinraza (nusinersen) in England, after the…

Destiny McCalvin gave birth to her second child, a girl she named Kenzlee, after what she and her doctors took to be an uneventful pregnancy in 2011. “She seemed like a normal baby; she weighed 7 pounds 7 ounces, no issues,” her mother said. But “issues” soon became clear: “It…

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…

Treating adults with spinal muscular atrophy (SMA) with Spinraza (nusinersen) was safe and improved their muscle strength, and motor and respiratory function, among other benefits, according to preliminary research. While treatment with Biogen‘s Spinraza has been demonstrated to be beneficial in children and infants with SMA,…

Risdiplam continues to show promise as a therapy for individuals with spinal muscular atrophy (SMA) types 2 and 3, results from an ongoing Phase 2/3 trial show. Results from the study, “Update from SUNFISH Part 1: Safety, Tolerability and PK/PD from the Dose-Finding Study, Including…

Risdiplam (RG7916) can improve both survival and motor function in patients with type 1 spinal muscular atrophy (SMA) beyond that expected in the natural history of the disease, according to recent clinical data. These data were presented during the 2019 annual meeting of the American Academy of…

A single administration of Zolgensma continues to extend the survival of patients with spinal muscular atrophy (SMA) type 1 while halting motor neuron loss several years after dosing, according to long-term clinical data. Zolgensma, developed by AveXis — later purchased by Novartis in 2018 — is a one-time treatment intended…

Spinal muscular atrophy (SMA) treatment with Biogen’s Spinraza (nusinersen) has resulted in motor milestone achievements in line with normal motor development and continues to provide benefit to infants who began receiving the treatment in a presymptomatic stage of the disease, according to interim clinical trial results. The study, “…

Treatment with Spinraza (nusinersen) results in greater improvements in motor function and continues to improve or stabilize motor function scores in spinal muscular atrophy (SMA) patients, according to recent clinical data. The findings were presented during the 2019 American Academy of Neurology (AAN) Annual Meeting, being…