After two years of treatment with Evrysdi (risdiplam), most babies with spinal muscular atrophy (SMA) type 1 in…
Articles by Marisa Wexler, MS
Continued treatment with apitegromab, designed to strengthen muscles, led to stabilization or improvement in measures of fatigue and life…
Children with spinal muscular atrophy (SMA) switching from Spinraza (nusinersen) to Evrysdi (risdiplam) are generally optimistic about…
Levels of an immune protein called CHIT1 change over time in children with spinal muscular atrophy (SMA) who undergo…
Treatment with Evrysdi (risdiplam) increased levels of SMN protein and stabilized motor function in a Phase 2 clinical trial…
The gene therapy Zolgensma (onasemnogene abeparvovec-xioi), approved in the U.S. for very young children with spinal muscular atrophy…
Doctors need to be well-educated about options in the evolving treatment landscape to help patients with spinal muscular atrophy…
Note: This story was updated Sept. 19, 2022, to correct that 78.9% of the survey respondents would choose an…
In its final decision, the Canadian Agency for Drugs and Technologies in Health (CADTH) recommended against Spinraza (nusinersen)…
Prescribing information for Zolgensma (onasemnogene abeparvovec-xioi), an approved gene therapy for spinal muscular atrophy (SMA), is being updated…