#AANAM – Neurofilaments Before Spinraza May Predict Motor Improvements
Higher blood levels of phosphorylated neurofilament heavy chain (pNF-H) — a marker of nerve cell damage — before beginning Spinraza…
Articles by Marta Figueiredo, PhD
Prenatal Enrollment Yields Faster Results from Newborn Screening
Prenatal enrollment of newborns for spinal muscular atrophy (SMA) screening leads to faster results than recruitment after birth, according to…
Immune Response to Zolgensma Can Be Common in Older SMA Children
Abnormal increases in liver enzymes levels — suggestive of liver damage — and drops in platelet counts are common among…
#AANAM – Type 1 Infants Using Evrysdi for 2 Years Gaining in Skills
Evrysdi (risdiplam) continues to improve survival and to allow infants with spinal muscular atrophy (SMA) type 1 to achieve key motor milestones, according…
Apitegromab Over 1 Year Bolsters Motor Abilities of Types 2 and 3: TOPAZ Trial
Apitegromab, Scholar Rock’s muscle-directed therapy for spinal muscular atrophy (SMA), safely and effectively improved or stabilized motor function in children and…
Evrysdi Approved in Europe as First Oral, At-home Treatment
The European Commission has approved Evrysdi (risdiplam) as the first oral and at-home treatment for adults, children, and infants 2 months…
Survey: Benefits of SMA Newborn Screening Outweigh Disadvantages
The benefits of newborn screening (NBS) for spinal muscular atrophy (SMA) outweigh its disadvantages and challenges, according to a…
#MDA2021 – Zolgensma Helped Pre-symptomatic Babies Achieve Age-Appropriate Motor Milestones
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference,…
#MDA2021 – Zolgensma Continues to Prevent Motor Function Decline for 5 Years
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference,…
#MDA2021 – Evrysdi Leads to Longer-term Benefits for SMA Types 2, 3, Data Show
Editor’s note: The SMA News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference,…