José Lopes, PhD,  managing science editor—

Zolgensma (onasemnogene abeparvovec-xioi), the newly approved gene therapy to treat SMA in infants and children under 2 years old, works by delivering directly to motor neurons a healthy copy of the SMN1 gene that is damaged by mutations in these patients and unable to make an essential protein. Carried…

Zolgensma, a first gene therapy for spinal muscular atrophy — and first for any chronic neurologic disease — is now an approved and potential “one-time” intravenous treatment for pre-symptomatic newborns through 2-year-olds with any type of SMA, the U.S. Food and Drug Administration (FDA) announced today, issuing an historic decision.

Treatment with the experimental oral therapy risdiplam has been enabling patients with spinal muscular atrophy (SMA) to achieve key development milestones and clinically meaningful motor improvements beyond the natural history of the disease. Susan Begelman, MD, vice president, Neurology, Nonmalignant Hematology, Influenza, U.S. Medical Affairs at Genentech,…

Long-term results show Spinraza (nusinersen) is of unprecedented benefit to a broad range of spinal muscular atrophy (SMA) patients, said Wildon Farwell, executive director of clinical development at Biogen, which markets this approved treatment. With clinical development that spans “presymptomatic infants, to symptomatic infants, to symptomatic children,” Spinraza has “demonstrated…

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…

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…

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…

Treatment of presymptomatic babies with the investigational gene therapy Zolgensma provides rapid improvement in motor function in infants with spinal muscular atrophy (SMA) types 1, 2 and 3, according to preliminary results of a Phase 3 trial. The study, named SPR1NT (NCT03505099), is enrolling participants…

Type 2 patients with spinal muscular atrophy (SMA) given the gene therapy Zolgensma via spinal canal injection are showing no safety concerns so far and notable motor milestones, early results of a Phase 1 trial report. Based on these interim but promising data, AveXis anticipates a request being made for the…

SMN protein deficiency in spinal muscular atrophy (SMA) may lead to gastrointestinal (GI) dysfunction by shrinking the smooth muscle in the bladder and altering specific cells in the colon, according to a mouse study. The study, “Smooth muscle atrophy and colon pathology in SMN deficient mice,”…