News

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),…

Olesoxime, a small molecule once considered a potential treatment, failed to slow motor and respiratory decline in people with spinal muscular atrophy (SMA) types 2 and 3 who were unable to walk, according to final results from an extension Phase 2 clinical trial. Data covering nearly 2.5 years of treatment…

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…

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…

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…

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…

By taking advantage of a natural process of gene silencing, a new gene therapy approach appears to prevent the toxicity to dorsal root ganglion (DRG) — a specific cluster of sensory neurons — seen in non-human primates during gene therapy studies for neurological disorders, researchers report. The approach successfully protected…

Repeated administrations of Spinraza (nusinersen) were associated with the presence of macrophages — immune cells specialized in recognizing and eliminating harmful organisms and dead cells — with unusual features in the spinal fluid of two infants with spinal muscular atrophy (SMA), a case study reports. While these…

Evrysdi (risdiplam) at its approved and therapeutic dose does not lead to eye damage in children or adults with spinal muscular atrophy (SMA), data from extensive ophthalmologic monitoring of patients in its clinical trials — prompted by earlier safety findings in monkeys — show. These data support the therapy’s favorable…