The first patient has been dosed in a Phase 4 trial exploring the safety and potential benefits of Spinraza (nusinersen) in children with spinal muscular atrophy (SMA) who failed to respond adequately to Zolgensma. The two-year study, called RESPOND (NCT04488133), was launched last year by…
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The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for a…
Cure SMA has developed a new three-part webinar series that delves into health and wellness topics pertinent to the spinal muscular atrophy (SMA) community. The three monthly installments of the Wellness Webinar Series will teach participants about a variety of strategies and tools that can be used…
After finding no safety concerns in Part A of the DEVOTE trial, which is testing higher doses of Spinraza (nusinersen) among people of all ages with spinal muscular atrophy (SMA), investigators are now screening patients for the study’s pivotal Part B, according to Biogen, the therapy’s maker. Part B,…
SMA News Today brought you daily coverage of the latest scientific findings, treatment developments, and clinical trials related to spinal muscular atrophy (SMA) throughout 2020. We look forward to reporting more news to patients, family members, and caregivers dealing with SMA during 2021. Here are the top 10 most-read articles…
Non-professional caregivers who care for children with spinal muscular atrophy (SMA) carry a high daily burden that negatively affects certain aspects of their lives, according to a questionnaire study conducted in Europe. Additionally, more severe disease was correlated with more time…
Some children with spinal muscular atrophy (SMA) may develop thrombotic microangiopathy (TMA) — a rare condition characterized by blood abnormalities — following treatment with Zolgensma, a study reports. A serious complication, TMA was resolved with supportive…
Health Canada has approved Novartis‘ gene therapy Zolgensma (onasemnogene abeparvovec) to treat pediatric spinal muscular atrophy (SMA) patients who have three or fewer copies of the SMN2 gene, or with infantile-onset disease. “Today’s announcement about the Canadian approval of Zolgensma is a significant milestone in our journey to reimagine medicine…
Administering Spinraza (nusinersen) via a particular method of injection, called the paramedian approach, can shorten procedure times and reduce the occurrence of adverse events for people with spinal muscular atrophy (SMA), a small study suggests. This method may prove to be a much easier route of administration for…
Direct medical costs associated with spinal muscular atrophy (SMA) are more than 50 times higher than those of matched control patients without SMA, according to a healthcare claims analysis. The primary driver of cost for infantile SMA cases was inpatient expenses,…
