Motor Function, Breathing Important in Choosing Treatment
Improvement in motor function, breathing, and ease of administration are among the factors that people with spinal muscular atrophy…
Articles by Marisa Wexler, MS
Cure SMA Booklet Outlines Possible Risks, Benefits of Combining Treatments
A free resource booklet, published by Cure SMA, may help people with spinal muscular atrophy (SMA) to better understand the…
Zolgensma Approved to Treat Young SMA Patients in Canada
Health Canada has approved Novartis‘ gene therapy Zolgensma (onasemnogene abeparvovec) to treat pediatric spinal muscular atrophy (SMA) patients…
Paramedian Injection Approach Found to Ease Spinraza Administration
Administering Spinraza (nusinersen) via a particular method of injection, called the paramedian approach, can shorten procedure times and reduce…
Spinraza’s Benefits Mild, Transient in Infant With Severe Type 0 SMA
An infant with type 0 spinal muscular atrophy (SMA), a severe disease form evident before birth, began treatment with…
NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Risdiplam, Oral SMA Treatment, Up for Approval in Japan
Chugai Pharmaceutical, part of the Roche Group, is asking that risdiplam be approved as a daily oral treatment for …
Adults With SMA Who Sought Specialty Care After Spinraza Approval May Have More Severe Disease
Adults with spinal muscular atrophy (SMA) who began being seen at specialty centers after the approval of Spinraza…
NICE Widens Its Zolgensma Appraisal Due to European Marketing Authorization
England’s National Institute for Health and Care Excellence (NICE) is expanding its appraisal of Zolgensma, a gene therapy for…