STAT3d
Novartis finally delivers gene therapy data in older SMA patients. Will they still want it?
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
BioPharma Dive2d
Novartis builds case for new SMA gene therapy
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
Zacks Investment Research on MSN2d
Novartis Reports Updated Positive Data From Phase III SMA Program
Novartis NVS reported promising new safety and efficacy results from its phase III studies of investigational intrathecal ...
Fierce Pharma3d
Novartis details intrathecal Zolgensma data in bid to reach more SMA patients with gene therapy
A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...
Nasdaq3d
Novartis' Gene Replacement Therapy For SMA Demonstrates Meaningful Efficacy And Safety Results
with the aim of offering OAV101 IT as a one-time treatment option to SMA patients. If regulatory filings be successful, Novartis anticipates the potential for this investigational gene therapy to ...
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 ...
Clinical Trials Arena on MSN2d
Novartis reports Phase III efficacy and safety outcomes of SMA treatment
In this trial, the efficacy and safety of the therapy were evaluated against a sham control in treatment-naïve SMA Type 2 ...
Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
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 ...
Yahoo Finance2d
Novartis builds case for new SMA gene therapy
Key to that mission is the “STEER” study, a late-stage trial of children with moderate SMA who are older than ... major concern and focal point for gene therapy developers.