Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), was generally safe and showed dose-dependent accumulation and clearance of the inactive form of myostatin, a muscle growth suppressor, in healthy volunteers, according to data from a Phase 1 clinical trial. These findings, published in the journal Advanced…
Cure SMA is offering free “independence assistance packages” to teenagers and adults with spinal muscular atrophy (SMA) in the U.S. to assist with everyday living or to help make living on their own possible. The Teen & Adult Independence Assistance Package is meant to supplement the nonprofit organization’s…
The blood levels of neurofilaments — a marker of nerve cell damage — may serve as a disease onset and treatment response biomarker in infants with spinal muscular atrophy (SMA) type 1, but not in older patients with milder disease forms, a study suggests. These findings add to previous studies…
Novartis Gene Therapies has added thrombotic microangiopathy (TMA) — a rare condition characterized by blood abnormalities — to the list of safety concerns with use of Zolgensma in children with spinal muscular atrophy (SMA). This decision was driven by reports of six confirmed cases of TMA within one to two…
Sex and the number of copies of the “backup” SMN2 gene influence both the age of symptom onset and the risk of losing the ability to walk in people with spinal muscular atrophy (SMA) type 3, according to a Polish registry study. The data also confirmed the notion…
A decision from England’s National Institute for Health and Care Excellence (NICE) will make Spinraza (nusinersen) available to people with spinal muscular atrophy (SMA) type 3 who are unable to walk. In England, Spinraza is available through a managed access agreement (MAA) between NICE, the National Health…
Newborn screening can aid in the early diagnosis and prompt treatment of spinal muscular atrophy (SMA), which can lead to better clinical outcomes, according to a German study. “We strongly recommend that newborn screening for SMA ought to become universal in countries that provide SMA-specific medication,” the study’s researchers…
A researcher from Keele University School of Medicine in the U.K. was awarded a £99,959 ($138,633) research grant to identify and evaluate potential — and more affordable — treatments to improve the health of children with spinal muscular atrophy (SMA). The two-year research grant of the Academy of Medical…
Novartis will initiate SMART, a Phase 3b clinical study to evaluate the safety and efficacy of the one-time gene therapy Zolgensma(onasemnogene abeparvovec) in young children with spinal muscular atrophy (SMA) who weigh 8.5 to 21 kilograms (about 18 to 46 pounds). Zolgensma is approved in the U.S.
Ayah Lundt, a 15-month-old Danish girl with spinal muscular atrophy (SMA) type 2, is helping bring the world together as her parents race to raise $2.4 million for a single dose of Zolgensma before she…
