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The gene therapy Zolgensma safely improved mobility in young children with spinal muscular atrophy (SMA) living in Qatar, a real-world study shows. The case series study, “Gene therapy for spinal muscular atrophy: the Qatari experience,” was published in the journal Nature Gene Therapy. Zolgensma…
The U.S. Food and Drug Administration (FDA) has lifted its partial hold on clinical trials of OAV-101 (AVXS-101), an investigational intrathecal gene therapy for the treatment of spinal muscular atrophy (SMA). The agency put a hold on the open-label clinical trial STRONG in 2019, citing concerns seen in…
Daily treatment with Evrysdi (risdiplam) following the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and leads to motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. While children were followed for a short time after Evrysdi was…
More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…
From a Zoom book discussion to candle lightings, efforts to mark Spinal Muscular Atrophy Awareness Month, observed each August, are underway to increase awareness of this rare genetic disease and raise funds to battle it. Supporters are involved in a range of activities that including swapping out their social…
To better understand and address barriers to the implementation of newborn screening across Canada for spinal muscular atrophy (SMA), Muscular Dystrophy Canada (MDC) has awarded more than $700,000 to projects in three provinces. This is the first round of awards to come from a collaboration between MDC…
Spinraza (nusinersen), a treatment for spinal muscular atrophy (SMA) that helps improve motor function, was found to also improve lung function in a 10-year-old girl with advanced SMA type 2, a case report says. The report, “Nusinersen improved respiratory function in spinal muscular atrophy type 2,”…
The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…
Novartis is stopping the clinical development of branaplam (LMI070), its experimental oral treatment for spinal muscular atrophy (SMA) being evaluated in a Phase 1/2 clinical trial. According to the company’s announcement, this “difficult” decision was based on the “rapid advancements in the SMA treatment landscape in recent years,”…
A series of toxicology studies in rats and macaque monkeys confirmed the safety and tolerability of apitegromab, an investigational spinal muscular atrophy (SMA) therapy, supporting its ongoing assessment in patient trials, a study reported. The study, “Preclinical Safety Assessment and Toxicokinetics of Apitegromab, an Antibody Targeting…
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