Olesoxime Failed to Benefit SMA Type 2 and 3 Patients, Final Trial Data Show
Olesoxime, a small molecule once considered a potential treatment, failed to slow motor and respiratory decline in people with spinal…
Articles by Marta Figueiredo, PhD
Gene Therapy Given Directly to Spinal Canal Might Be Safer With ‘Silencing’ Step
By taking advantage of a natural process of gene silencing, a new gene therapy approach appears to prevent the…
Spinraza Shown to Preserve Respiratory Muscle Strength in SMA Type 2 Patients
Spinraza (nusinersen) effectively preserves respiratory muscle strength in addition to motor function in children with spinal muscular atrophy (SMA) type 2,…
Molecule Similar to Evrysdi But Possibly Safe at Higher Doses Identified
A newly identified small molecule able to cross into the brain — named TEC-1 — significantly eased disease severity and…
Screening for Carriers of SMA May Be Cost-free Option for Couples in Australia
The Australian Medical Services Advisory Committee (MSAC) has recommended that the costs of screening for mutations known to cause …
NICE Reviews UK Spinraza Reimbursement for SMA Type 3 Patients Unable to Walk
England’s National Institute for Health and Care Excellence (NICE) has begun a review process to decide whether to include spinal muscular…
Muscle-directed Therapy SRK-015 Improves Motor Function in SMA Types 2, 3, Data Show
SRK-015, Scholar Rock’s muscle-targeted therapy, safely and effectively improved motor function in children and young adults with…
SMA Type 3 Adults May Have Issues Regulating Component of Cell’s ‘Skeleton,’ Study Suggests
Adults with spinal muscular atrophy (SMA) type 3 have significantly lower levels of several molecules that help regulate actin,…
Spinraza Leads to Motor Gains Over Time in SMA Type 3 Adults, Real-Life Study Finds
About one year of treatment with Spinraza (nusinersen) gradually and significantly improves motor function in adults with spinal…
Evrysdi Open to Eligible Patients in UK Through Early Access Program
Evrysdi (risdiplam), Roche’s oral therapy for spinal muscular atrophy (SMA), will be available to select patients in…