More frequent participation in social and leisure activities is associated with less caregiver burden and emotional distress for mothers of patients with spinal muscular atrophy (SMA), according to a Dutch study. The research, “Participation and mental well-being of mothers of home-living patients with spinal muscular atrophy,”…
Use of speech-generating devices (SGDs) improves the quality of life of children with spinal muscle atrophy (SMA) type 1 through better communication skills, greater independence, and personal relationships, according to the results of a survey of parents. The study, “Communication skills among children with spinal muscular atrophy…
A new gene therapy approach using RNA molecules called transfer RNAs (tRNAs) was able to repair a subset of mutations causing spinal muscular atrophy (SMA) and other inherited diseases in living muscle tissue of mice, a study shows. These genetic alterations, called nonsense mutations, alter the DNA…
The U.S. Food and Drug Administration approved the six-minute walk test (6MWT) as an acceptable primary endpoint, or goal, for a potential registration program of the investigational treatment reldesemtiv for spinal muscular atrophy (SMA) patients who are able to walk. In the feedback provided to therapy developer Cytokinetics, the agency…
A preliminary Draft Evidence Report from the Institute for Clinical and Economic Review (ICER) says the investigational spinal muscular atrophy (SMA) treatment Zolgensma (onasemnogene abeparvovec-xxxx) could be more cost-effective than Spinraza (nusinersen) with a one-time price of $2 million. However, ICER noted that more must…
SMA News Today brought you daily coverage of important findings, key treatment developments and clinical trials related to spinal muscular atrophy (SMA) throughout 2018. We look forward to reporting more news that is important for the SMA community during 2019. Here are the top 10 most-read SMA articles of 2018, with…
An increased amount of SMN protein — crucial to the survival of motor neurons — in the blood could reflect similar increases in the brain, spinal cord, and peripheral tissues after spinal muscular atrophy (SMA) treatment with the oral therapy risdiplam, researchers say. They believe these findings in animal…
Treatment of spinal muscular atrophy (SMA) with Spinraza (nusinersen) through the spinal canal is feasible and leads to high patient compliance as long as it’s done under the supervision of an experienced multidisciplinary team, according to a single-center study in Italy. The study, “Intrathecal nusinersen…
The U.S. Food and Drug Administration (FDA) has granted priority review to Novartis’ Biological License Application (BLA) asking that Zolgensma (onasemnogene abeparvovec-xxxx) — previously called AVXS-101 — be approved as a gene therapy for spinal muscular atrophy (SMA) type 1. Its request is reported to specifically cover intravenous…
Novartis may put a $4 million to $5 million price tag on its “one-time, potentially curative” gene therapy AVXS-101 for spinal muscular atrophy (SMA) and believes this could be cost-effective. “Four million dollars is a significant amount of money, but we believe that this is a cost-effective point,”…
