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 …
FDA Approves Gene Therapy Onasemnogene Abeparvovec for ...
Nov 25, 2025 · Novartis receives FDA approval for Itvisma, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA). News release. …
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 muscular …
Muscular Dystrophy Association Calls FDA Approval of Novartis ...
Nov 24, 2025 · Muscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy …
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 mutation of the …
Novartis reports Phase III outcomes of SMA treatment
Mar 20, 2025 · Novartis has reported positive efficacy and safety outcomes from its Phase III clinical programme for OAV101 IT (onasemnogene abeparvovec), an investigational intrathecal therapy for …
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 significant …
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 …
Intrathecal Zolgensma Meets Primary End Point in Phase 3 ...
Jan 2, 2025 · In newly announced data from the phase 3 STEER study (NCT05089656), results revealed that OAV101IT (Novartis), an intrathecal (IT) investigational formulation of an approved …