Author Archives: Marisa Wexler MS

Among adults with spinal muscular atrophy (SMA) being treated with Spinraza (nusinersen), a decrease in levels of the inflammatory protein YKL-40 in the fluid around the brain is associated with improvements in arm and hand function, according to a new study. “We conclude that YKL-40 in CSF…

The RNA-binding protein Mid1 increases levels of the mutated androgen receptor (AR) that drives nerve cell degeneration in spinal and bulbar muscular atrophy (SBMA), a new study shows. Since Mid1 is expressed mainly in lower motor neurons, the nerve cells mainly affected in SBMA, the study’s researchers proposed…

No new safety signals related to Evrysdi (risdiplam) were documented in a compassionate use program in Germany where more than 100 people with spinal muscular atrophy (SMA) were treated with the oral therapy prior to its approval last year, a new study reports. “We present the first…

Spinraza (nusinersen) treatment was generally well tolerated and led to improved or stabilized motor function for nearly 300 children with spinal muscular atrophy (SMA) in Poland, a new study reports. The study, “Safety, tolerability, and efficacy of a widely available nusinersen program for Polish children with Spinal…

Pediatric neurologists who are more comfortable with ambiguity are more likely to make suboptimal treatment decisions for their patients with spinal muscular atrophy (SMA), according to a new study from Spain. “Our results may contribute to the growing evidence of the relevance of value-based shared decision-making for the current…

The total medical costs associated with spinal muscular atrophy (SMA) are lower for patients diagnosed at birth via newborn screening, compared with those diagnosed after they start showing symptoms of the disease, a new study has found. These findings “demonstrate clearly that as long as the decision to reimburse…

The newborn screening program at Cadham Provincial Laboratory in Manitoba, Canada, is expanding to include spinal muscular atrophy (SMA), according to an announcement from Audrey Gordon, the province’s health minister. “Early diagnosis is the key to improving outcomes for babies born with metabolic, endocrine or genetic disorders such as…

Among adults with spinal muscular atrophy (SMA) type 3, those with greater motor impairment tend to score better on some cognitive measures, but worse on others, a new study reports. Among those with SMA, “patients with greater motor difficulties had lower performance in attention, but higher performance in measures…

The U.S. Food and Drug Administration (FDA) has extended its approval of Evrysdi (risdiplam) to treat babies with spinal muscular atrophy (SMA) who are younger than 2 months old. Evrysdi, an oral therapy that works by increasing the levels of the survival motor neuron (SMN) protein that is missing in…

A committee of the Canadian Agency for Drugs and Technologies in Health (CADTH) has issued a draft recommendation advising Spinraza (nusinersen) should not be reimbursed for the treatment of adults with spinal muscular atrophy (SMA).  CADTH is an independent, nonprofit organization that provides evidence-based advice to…