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 therapeutic choice,” according to Angela Lek, PhD, chief research officer at the Muscular…
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A new review of studies from six countries reveals that incorporating spinal muscular atrophy (SMA) into newborn screening (NBS) programs yields clinical improvements and reduces healthcare spending, particularly when paired with presymptomatic treatment. “These results collectively indicate that NBS for SMA, when combined with presymptomatic treatment, is not only…
Mifepristone, a glucocorticoid blocker, increased survival and muscle size in a mouse model of spinal muscular atrophy (SMA), according to a recent study. Combining mifepristone with a gene therapy construct similar to the one used in Zolgensma and Itvisma, which is intended to increase levels of…
Treatment with Zolgensma (onasemnogene abeparvovec-xioi) improved motor function in children with spinal muscular atrophy (SMA) who needed tracheostomy, without posing new safety risks, a real-world study showed. Tracheostomy is a surgical procedure that creates an opening in the windpipe, often necessary when a ventilator is used to assist…
Fatigue in the diaphragm — the large muscle at the base of the chest that pulls air in and out of the lungs — may contribute to difficulty breathing in people with spinal muscular atrophy (SMA), a new study reports. The study did not find evidence of fatigue in…
Last week’s U.S. Food and Drug Administration (FDA) approval of Novartis‘ gene therapy Itvisma (onasemnogene abeparvovec-brve) means one-time gene therapy is now authorized for all people with spinal muscular atrophy (SMA) in the U.S., an “important moment” for the SMA community, said Tracey Dawson, PhD, senior vice…
The specialty pharmacy Orsini was selected as a partner to provide Itvisma (onasemnogene abeparvovec-brve), the newly approved gene therapy for people with spinal muscular atrophy (SMA), ages 2 and older. Itvisma, marketed by Novartis, is the first gene therapy approved in the U.S. for older children and…
Children with spinal muscular atrophy (SMA) have higher rates of emotional and behavioral challenges than children in the general population, even with today’s modern treatments, a new study reports. “In our treated SMA cohort, mental health challenges occurred across a variety of severities of motor function and created an…
Before the end of this year, thanks to a new decision by the U.S. Food and Drug Administration (FDA), one-time gene therapy will be available to people of all ages with spinal muscular atrophy (SMA) in the United States. The first SMA gene therapy, Zolgensma (onasemnogene abeparvovec-xioi), was…
A high-dose regimen of nusinersen — the active ingredient in Spinraza — has been recommended for approval to treat spinal muscular atrophy (SMA) by the Committee for Medicinal Products for Human Use (CHMP), a branch of the European Medicines Agency. The decision was based on positive data from…
