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 …
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 …
Novartis secures approval from FDA for Itvisma to treat SMA
Nov 25, 2025 · Novartis has received approval from the US Food and Drug Administration (FDA) for the Itvisma (onasemnogene abeparvovec-brve) gene replacement therapy to treat spinal …
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 …
FDA Approves Novartis Gene Therapy Itvisma for Rare Muscle ...
Nov 25, 2025 · Novartis Gene Therapy: The FDA has granted approval for Novartis' gene therapy Itvisma, designed for spinal muscular atrophy in patients two years and older, marking a …
FDA approves Itvisma in spinal muscular atrophy for patients ...
5 days ago · 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 …
One-Time Gene Therapy Itvisma Approved for Spinal Muscular ...
2 days ago · The FDA approved Itvisma gene therapy for patients aged 2 years and older with spinal muscular atrophy and confirmed SMN1 gene mutation.
Cure SMA Welcomes Broad FDA Approval of Itvisma™ for Spinal ...
Nov 26, 2025 · Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease that affects the motor nerve cells in the spinal cord and impacts the muscles used for activities …
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 …
Novartis' Gene Replacement Therapy For SMA Demonstrates ...
Mar 19, 2025 · Novartis AG (NVS), Wednesday, announced the positive results from its Phase III clinical program of investigational gene replacement therapy, intrathecal onasemnogene …