Spinal Muscular Atrophy (SMA) - Novartis
Spinal Muscular Atrophy (SMA) SMA is a rare and devastating genetic disease caused by a lack of a functional survival motor neuron 1 (SMN1) gene, resulting in the rapid and irreversible loss of motor …
Novartis peels back Zolgensma IT data in older SMA patients
Mar 19, 2025 · A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the treatment of older patients with spinal muscular atrophy …
Zolgensma-Like Gene Therapy Benefits Older Spinal Muscular ...
3 days ago · A different formulation of Novartis’s gene therapy Zolgensma (onasemnogene abeparvovec), known as Itvisma, achieved good results in a Phase III trial in spinal muscular atrophy …
FDA approves Itvisma in spinal muscular atrophy for patients ...
Dec 2, 2025 · The FDA has approved an adeno-associated virus 9 gene replacement therapy to treat individuals aged 2 years and older with spinal muscular atrophy and a confirmed mutation of the …
FDA approves Novartis' Itvisma gene therapy for SMA - NJBIZ
Dec 3, 2025 · Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 and older.
Novartis Shares Positive Phase III Data for Spinal Muscular ...
Mar 20, 2025 · Novartis has announced positive results from its Phase III clinical trial program evaluating intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement …
Novartis exec: Itvisma approval 'important moment' for community
5 days ago · Last week’s U.S. Food and Drug Administration (FDA) approval of Novartis ‘ gene therapy Itvisma (onasemnogene abeparvovec-brve) means one-time gene therapy is now authorized for all …
Intrathecal onasemnogene abeparvovec in treatment-naive ...
3 days ago · The phase 3 STEER trial showed that a single intrathecal dose of onasemnogene abeparvovec significantly improved motor function in children and adolescents with spinal muscular …
Novartis’ Phase III spinal muscular atrophy therapy trial ...
Dec 31, 2024 · Novartis has reported positive topline outcomes from the Phase III STEER trial of an investigational gene therapy, intrathecal onasemnogene abeparvovec (OAV101 IT) in treating …
Phase III Trial Shows Novartis’ Gene Therapy Significantly ...
Mar 21, 2025 · Results from the STEER and STRENGTH studies showed that OAV101 IT led to a 2.39-point improvement in motor function in patients with spinal muscular atrophy. Results from the Phase …