News

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…

New treatments for spinal muscular atrophy (SMA) are helping many people live longer, fuller lives, changing what it means to grow up — and grow older — with the condition. SMA is now being managed across all stages of life, shifting care from palliative support toward long-term health and…

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…