News

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,…

Researchers have found changes in the electrical activity between the brain and muscles in adults who had childhood polio — an infection that affects the same motor neurons as those impacted in people with spinal muscular atrophy (SMA), a…

Genetic analysis of 28 families with a member affected by spinal muscular atrophy (SMA) identified novel mutations in the SMN1 gene and different genes widely prevalent in SMA patients not found in unaffected family members, a study reveals. …

Most children with spinal muscular atrophy (SMA) show an abnormal increase in the levels of liver enzymes — suggestive of liver damage — following treatment with Zolgensma, according to a study by Novartis, the therapy’s developer. This known side effect of the gene therapy was mainly very mild and…

The Muscular Dystrophy Association (MDA) is encouraging the U.S. Centers for Disease Control and Prevention(CDC) to recommend that people living with neuromuscular diseases (NMDs) have early access to any federally approved COVID-19 vaccine. The MDA made its request in a letter to members of the CDC…

A Neurological Alliance report is calling for improved treatment and care for the 150,000 U.K. residents living with rare neurological conditions such as spinal muscular atrophy (SMA). The report presents what the advocacy organization believes should be included in the U.K.’s coming new governmental framework for…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…