News

Spinraza May Restore Development of Motor Neurons in SMA Children

Spinraza (nusinersen) may restore the normal development of motor neurons — the nerve cells responsible for making muscle cells work — in children with spinal muscular atrophy (SMA), particularly those who start treatment early, a study found. Besides getting motor neurons back onto their developmental trajectory, Spinraza also may slow their…

Funding Bolsters Effort to Bring Newborn Screening to Eastern Canada

A CA$414,000 grant was awarded to the Maritimes by Muscular Dystrophy Canada (MDC) and Novartis Pharmaceuticals Canada to further support the implementation of newborn screening for spinal muscular atrophy (SMA). The funding (about $322,600), received by the Maritime Newborn Screening Program (MNSP), will go toward work to…

Real Life Inspires Son With SMA on ‘Ordinary Joe’ TV Show

One of NBC’s newest TV shows, “Ordinary Joe,” is making a mark in the spinal muscular atrophy (SMA) community for including a character with the disease. The drama series follows protagonist Joe Kimbreau, played by James Wolk, through three separate realities stemming from a single decision he made…

Saliva Samples Can Help Diagnose SMA, Study Demonstrates

Genetic analysis of dried saliva spots identified people with spinal muscular atrophy (SMA), a study demonstrated and possibly for the first time. The scientists noted that this noninvasive and low-cost, alternate method of genetic screening may be suitable for programs held in schools or workplaces without the need for…

Poor Spinraza Adherence Leads to More Illness, Healthcare Costs

Not having Spinraza (nusinersen) therapy as prescribed — called treatment non-adherence by clinicians — increased the frequency of co-existing diseases, required greater use of healthcare resources, and raised overall costs for patients with spinal muscular atrophy (SMA) in the U.S. and their families, retrospective research into claims data for…

Treatment Delays May Be Less Important Than Family Support

The COVID-19 pandemic led to delays in treatment with Spinraza (nusinersen) for many children with spinal muscular atrophy (SMA). However, these delays seem to have a lower impact on children’s functional skills when compared with family support, according to a small study in Italy. The study, “…