FDA Shifts Risdiplam Decision to August to Include Data on Older Patients
The U.S. Food and Drug Administration (FDA) has extended its review of risdiplam as an oral treatment for spinal…
Articles by Marta Figueiredo, PhD
Spinraza’s Way of Modifying ASOs Seen as More Effective in Mouse Model
A specific chemical modification of antisense oligonucleotides (ASOs) — called MOE; used in Spinraza (nusinersen) — was more…
Body Composition May Be Biomarker of Motor Function in SMA Patients, Study Suggests
Higher levels of free-fat body mass and lean body mass are associated with better motor function in children with…
Risdiplam Use ‘Meaningful’ to Broadest SMA Group Ever in Clinical Trial, Genentech Exec Says
Risdiplam treatment has led to “clinically meaningful” improvements — whether gains or disease stabilization — in the broadest range…
Risdiplam Leads to Motor Skills Gains or Stability in SMA Types 2 and 3, SUNFISH Trial Reports
Risdiplam safely and significantly improved or stabilized motor function in children and young adults with spinal muscular atrophy (SMA)…
Researchers Develop Low-Cost, Reliable Method for Newborn Screening for SMA
Researchers in China report having developed a method that was fast, effective, and reliable in newborn screening (NBS) for…
CSF Protein Profile May Help Predict Spinraza Responses in Late-onset SMA Patients, Study Shows
Analysis of the protein levels in the cerebrospinal fluid (CSF) of people with late-onset spinal muscular atrophy (SMA) may…
ZPR1 Protein May Be New Potential Therapeutic Target for SMA, Preclinical Study Suggests
Increasing the amount of ZPR1 — a protein found in low levels in people with spinal muscular atrophy (SMA)…
Top 10 SMA Stories of 2019
SMA News Today brought you daily coverage of key findings, treatment developments — including in-depth coverage of Zolgensma’s…
Spinraza Stabilizes or Improves Motor Function in Older SMA Patients, US Real-world Study Suggests
Spinraza (nusinersen) safely stabilizes or improves motor function in older patients with spinal muscular atrophy (SMA), according to…