Three men with late-onset spinal muscular atrophy (SMA) undergoing treatment with the approved therapy Evrysdi (risdiplam) were able to conceive with their partners, a case series reported. None of the pregnancies were planned, and the researchers noted there were no missed treatment doses. Among the three, one pregnancy…
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A year of disease-modifying therapies (DMTs) improved motor function, particularly fine motor skills, in people with spinal muscular atrophy (SMA) who could sit, but not walk due to disease progression before they had access to treatment, a study reports. These functional improvements were independent of treatment-related gains in…
Powered, ride-on toy cars to help children with spinal muscular atrophy (SMA) type 1 be more mobile must be adapted for easy access and match a child’s motor abilities, according to parents who took part in a 12-week training program in Spain. The parents also said it’s important to…
Most adolescents and adults with spinal muscular atrophy (SMA) have normal sensory nerve function, even those with long-lasting disease, regardless of SMA type, a study in the Netherlands shows. Nerve conduction tests had abnormal results in about 6% of the patients, which was about what was seen in healthy…
Children living with spinal muscular atrophy (SMA) in the U.K. can switch treatments based on practical needs and preferences without experiencing unexpected disease progression, according to a study that drew on data from the SMA REACH UK database. “The availability of treatments in the United Kingdom has fundamentally…
The balance of amino acids capable of influencing neurological function is disrupted in patients with spinal muscular atrophy (SMA) and in mouse models, a study found. Observed changes in patients were partially normalized after treatment with the disease-modifying therapy Spinraza (nusinersen). Treatment with one of the amino acids,…
A tablet formulation of Evrysdi (risdiplam) for spinal muscular atrophy (SMA) recently approved in the U.S. is designed to give patients a new option, a Genentech official said. “Evrysdi is the only oral, non-invasive disease-modifying SMA treatment, and the new tablet formulation may provide greater freedom and…
Treatment with Spinraza (nusinersen) can help stabilize motor function in adults with spinal muscular atrophy (SMA), according to a new analysis. “This study aligns with previously reported findings and suggests that long-term [treatment with Spinraza] offers sustained clinical benefits in adults with SMA,” researchers wrote in the abstract…
It’s been almost six years since the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was first approved in the U.S. to treat young children with spinal muscular atrophy (SMA). Now, more than 95% of babies diagnosed with SMA are being treated with Zolgensma, according to Daniel Grant, vice president…
The muscle-strengthening therapy apitegromab is being considered for approval by the U.S. Food and Drug Administration (FDA) as an add-on treatment for spinal muscular atrophy (SMA), with a decision expected around the start of fall. Scholar Rock, the company developing apitegromab, applied seeking FDA…
