German Study Urges Newborn SMA Screening, Citing Better Outcomes
Newborn screening can aid in the early diagnosis and prompt treatment of spinal muscular atrophy (SMA), which can lead…
Articles by Marisa Wexler, MS
European Alliance Calls for Newborn Screening
The European Alliance for Newborn Screening for Spinal Muscular Atrophy is calling on all European states to introduce routine screening…
Spinal Fluid Changes With Spinraza’s Use Mild, Possibly Due to Lumbar Puncture
Treatment with Spinraza (nusinersen) may cause some alterations in the cerebrospinal fluid — that which surrounds the brain and…
Challenges of Life as Teen, Young Adult With SMA Highlighted in Survey
Being an adolescent or young adult with spinal muscular atrophy (SMA) presents unique challenges, according to a study based…
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…
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…