BioSpace4h
6 Therapies on the Horizon for Spinal Muscular Atrophy
Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...
Fierce Pharma4d
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 ...
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 ...
STAT4d
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 ...
SMA News Today4d
MDA 2025: SMA kids maintaining Zolgensma benefits 10 years later
Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...
Endpoints News4d
No­var­tis touts Zol­gens­ma da­ta in old­er kids as it read­ies reg­u­la­to­ry fil­ings
Novartis released two Phase 3 datasets on Wednesday that show a new version of its gene therapy Zolgensma led to motor ...
Precision Medicine Online3d
Novartis to Seek Approval for Intrathecal SMA Gene Therapy in Children, Young Adults in Early 2025
An intravenous form of the gene therapy is already approved and sold as Zolgensma in the US for patients less than 2 years of age.
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 ...
BioSpace2d
Novartis’ Fabhalta Approved as First Therapy for Rare Kidney Disease
This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal ...