Newborn Screening Pilot for SMA in Belgium Grows Into Official Program
A pilot newborn screening (NBS) program for spinal muscular atrophy (SMA) that started in Liège province has transitioned into an…
Articles by Marisa Wexler, MS
Phase 3 Apitegromab Trial Planned for Non-ambulatory Types 2, 3
Scholar Rock plans to soon initiate a Phase 3 clinical trial of apitegromab, its investigational muscle-directed treatment for …
Pre-symptomatic Infants Retain Swallowing Ability in Evrysdi Trial
After being treated with Evrysdi (risdiplam) for at least a year, pre-symptomatic infants with spinal muscular atrophy (SMA)…
Variant in Androgen Receptor Might Be Useful in Treating SBMA
A naturally occurring form of the androgen receptor protein — which scientists have named AR45 because of its…
SMA Caused by Mutation in ASAH1 Gene Reported in Romania
The first-ever case of someone in Romania with spinal muscular atrophy (SMA) caused by a mutation in the ASAH1…
FDA Lifts Hold on Clinical Trials of Intrathecal OAV-101
The U.S. Food and Drug Administration (FDA) has lifted its partial hold on clinical trials of OAV-101 (AVXS-101), an…
SMA Treatment May Be More Effective by Altering Underlying Molecule
Biochemical modifications made to antisense oligonucleotides — molecules that are the basis of an existing therapy for spinal muscular…
Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for…
SMA Added to North Carolina’s Newborn Screening Program
Note: This story was updated on June 21, 2021, to clarify that these genetic tests are after a baby’s…
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…