Author Archives: Marisa Wexler MS

UK NICE Expands Access to Spinraza for SMA Type 3

A decision from England’s National Institute for Health and Care Excellence (NICE) will make Spinraza (nusinersen) available to people with spinal muscular atrophy (SMA) type 3 who are unable to walk. In England, Spinraza is available through a managed access agreement (MAA) between NICE, the National Health…

German Study Urges Newborn SMA Screening, Citing Better Outcomes

Newborn screening can aid in the early diagnosis and prompt treatment of spinal muscular atrophy (SMA), which can lead to better clinical outcomes, according to a German study. “We strongly recommend that newborn screening for SMA ought to become universal in countries that provide SMA-specific medication,” the study’s researchers…

European Alliance Calls for Newborn Screening

The European Alliance for Newborn Screening for Spinal Muscular Atrophy is calling on all European states to introduce routine screening of all newborns for spinal muscular atrophy (SMA) by 2025. The recently-formed Alliance, which is composed of European patient organizations, academics, and the pharmaceutical industry, published its first white…

Motor Function, Breathing Important in Choosing Treatment

Improvement in motor function, breathing, and ease of administration are among the factors that people with spinal muscular atrophy (SMA) and their caregivers value most when deciding on available treatments, a new study reports. The study, “Treatment preference among patients with spinal muscular atrophy (SMA): a…

SMA Survey