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…

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…

Zolgensma (AVXS-101), an investigational gene therapy for patients with spinal muscular atrophy (SMA), improves motor function and prolongs survival in infants with SMA type 1 under six months of age, Phase 3 trial interim data show. These findings from the ongoing, multicenter and open-label Phase 3 STR1VE trial (…

Spinraza (nusinersen), an injectable medication approved two years ago by the U.S. Food and Drug Administration (FDA) to treat spinal muscular atrophy (SMA), is way overpriced. But so is its biggest potential competitor — Novartis‘ gene therapy Zolgensma — even though both have substantially helped children receiving either…

A preliminary Draft Evidence Report from the Institute for Clinical and Economic Review (ICER) says the investigational spinal muscular atrophy (SMA) treatment Zolgensma (onasemnogene abeparvovec-xxxx) could be more cost-effective than Spinraza (nusinersen) with a one-time price of $2 million. However, ICER noted that more must…

The U.S. Food and Drug Administration (FDA) has granted priority review to Novartis’ Biological License Application (BLA) asking that Zolgensma (onasemnogene abeparvovec-xxxx) — previously called AVXS-101 — be approved as a gene therapy for spinal muscular atrophy (SMA) type 1. Its request is reported to specifically cover intravenous…

Children with spinal muscular atrophy (SMA) are at a higher risk of hospitalization due to respiratory tract infections, including those caused by the respiratory syncytial virus (RSV), a small, single-center study in Switzerland suggests. Although 1 in 4 hospitalizations was due to an RSV infection, all the children with…

Nearly two-thirds of adults in South Korea with spinal muscle atrophy (SMA) type 2 or type 3 were initially misdiagnosed, according to a recent study. In type 2 cases, the primary misdiagnosis was a developmental delay, while muscular dystrophy was the main misdiagnosis in type 3…

Babies born prematurely were diagnosed with spinal muscular atrophy (SMA) through newborn screening programs (NBS) in Germany and, in most cases, started on treatment before disease symptoms emerged, a retrospective study reports. Doctors, however, waited to initiate treatment until many of the 12 newborns had reached full-term age,…