Pharmabiz1d
Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA
Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA: Basel Friday, March 21, 2025, 11:00 Hrs [IST] N ...
GlobalData on MSN2d
Novartis reports Phase III efficacy and safety outcomes of SMA treatment
Novartis has reported positive efficacy and safety outcomes from its Phase III clinical programme for OAV101 IT (onasemnogene ...
The Pharma Letter5d
Roche rounds off SUNFISH study with evidence of Evrysdi’s long-term efficacy
The Roche subsidiary Genentech has presented five-year SUNFISH data on Evrysdi (risdiplam) in the final readout of this ...
Novartis New Data From Single-Dose Gene Therapy Shows Meaningful Efficacy, Safety In Patients With Spinal Muscular Atrophy
Novartis' OAV101 IT gene therapy showed positive results in Phase 3 trials for SMA, with data supporting its potential as a ...
Applied Clinical Trials Online2d
Novartis Shares Positive Phase III Data for Spinal Muscular Atrophy Therapy
Intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement therapy, demonstrated a ...
Novartis announces results from Phase III program for OAV101 IT
Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene ...
Zacks.com on MSN2d
Novartis Reports Updated Positive Data From Phase III SMA Program
NVS reports new safety and efficacy data from the phase III program for its investigational OAV101 IT gene therapy for SMA ...
The Manila Times5d
Roche to share latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association (MDA) 2025 conference
Further information on the abstracts that Roche will present at MDA 2025 can be found below.
PMLive2d
Novartis shares positive phase 3 results for spinal muscular atrophy gene therapy
Novartis also shared results from the open-label phase 3b STRENGTH study of OAV101 IT in SMA patients aged two to less than 18 years who had discontinued treatment with nusinersen or risdiplam, which ...
The American Journal of Managed Care3d
STEER Data Open Door to SMA Gene Therapy for Wider Age Range of Children
Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...
Le Lézard6d
Scholar Rock Presents New Phase 3 SAPPHIRE Data at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
Scholar Rock , a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases where protein growth ...