Findlay boy provides comfort for Nationwide Children’s Hospital
He spent time at Nationwide Children’s Hospital. In November of 2023, Jackson was told he was going home. He thought this ...
SMA2h
Zolgensma into spinal canal drives SMA type 2 motor gains
The Phase 3 STEER trial enrolled more than 100 children with SMA type 2, ages 2-17, who were able to sit, but hadn't walked ...
FiercePharma4d
After delay with FDA, Novartis touts pivotal trial win for intrathecal Zolgensma in older SMA patients
More than three years after Novartis’ plan for a speedy approval for intrathecal Zolgensma was thwarted by an FDA requirement ...
Novartis Says Drug Helps Children With Spinal Atrophy in Study
Novartis AG said a new experimental medicine improved motor function in children with a form of spinal muscular atrophy, the ...
NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease
NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease This is the 2nd ...
Novartis’ Phase III spinal muscular atrophy therapy trial meets primary endpoint
Novartis has reported positive topline outcomes from the Phase III STEER trial of an investigational gene therapy, ...
FDA grants ODD to NMD Pharma’s Charcot-Marie-Tooth disease therapy
The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to NMD Pharma’s oral lead development ...
The Week8d
A Maldivian, born with spinal muscular atrophy, is beating all odds to pursue excellence
Typically, patients do not attend these meetings, with their family or caregivers representing them, says Unnikrishnan. But, ...
Monthly Prescribing Reference6d
Gene Therapy Improves Motor Function in Spinal Muscular Atrophy Type 2
The phase 3 STEER study enrolled 127 treatment-naïve patients aged 2 to less than 18 years with SMA type 2 who were able to sit but had never walked independently.
Novartis gene therapy helps children with rare muscle disorder in study
Swiss drugmaker Novartis said on Monday its gene therapy helped improve motor function in children with a rare muscle ...
MedPage Today7d
Pediatric Spinal Muscular Atrophy Peer-to-Peer
Newborn screening for spinal muscular atrophy allows for earlier diagnosis in preterm infants, leading to earlier use of disease-modifying therapies, in the presymptomatic stage, and better long ...