Author Archives: Marisa Wexler MS

Long-term treatment with Spinraza (nusinersen) led to clinically relevant improvements in motor function for most adults and older children with spinal muscular atrophy (SMA) in a study. “Our data provide real-world evidence for continuous effectiveness and safety of long-term [Spinraza] treatment in adults and older children regardless of…

Even though Ukraine has been at war for more than a year, a newborn screening program for spinal muscular atrophy (SMA) was launched late last year — and so far the program has successfully tested tens of thousands of babies, providing early diagnoses for nearly a dozen infants. A…

The European Commission has granted orphan drug status to taldefgrobep alfa as a potential treatment for spinal muscular atrophy (SMA). The designation is given to therapies that may improve care for rare diseases. It confers certain incentives, including a reduction in some regulatory fees and a guarantee of 10…

Treatment with Spinraza (nusinersen) can improve the movement ability of the diaphragm — the large muscle in the chest that helps pull air in and out of the lungs during breathing — in previously-untreated adults with spinal muscular atrophy (SMA), a study showed. The study, which was investigating…

For caregivers of children with spinal muscular atrophy (SMA), the availability of new disease-modifying treatments has offered hope — but difficulties in accessing these therapies and uncertainty about their long-term effectiveness also have fostered frustration and anxiety. That’s according to a new qualitative study, done in Canada, that…

For caregivers of children with spinal muscular atrophy (SMA), the availability of new disease-modifying treatments has offered hope — but difficulties in accessing these therapies and uncertainty about their long-term effectiveness also have fostered frustration and anxiety. That’s according to a new qualitative study, done in Canada, that aimed…

The Committee for Medicinal Products for Human Use (CHMP) favors European Union (EU) approval of Evrysdi (risdiplam) being extended to cover babies with spinal muscular atrophy (SMA) younger than 2 months of age. The recommendation now will be reviewed by the European Commission, which is expected to issue…

Measuring levels of neurofilament light chain (NfL) in the blood could be used to detect subtle nerve damage that may occur as a side effect of experimental treatments for diseases like spinal muscular atrophy (SMA). That’s according to the study, “Neurofilament Light Chain: A Translational Safety Biomarker…

Nonambulatory young adults with spinal muscular atrophy (SMA) who have contractures  — when muscle and connective tissue around a joint become short and hardened — do not feel significantly hindered by these in their daily lives. Despite contractures in multiple joints, they do not perceive them as a…

Nonambulatory young adults with spinal muscular atrophy (SMA) who have contractures  — when muscle and connective tissue around a joint become short and hardened — do not feel significantly hindered by these in their daily lives. Despite contractures in multiple joints, they do not perceive them as a constant…