In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

Novartis NVS reported promising new safety and efficacy results from its phase III studies of investigational intrathecal ...

Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...

Intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement therapy, demonstrated a ...

The challenge of the obesity epidemic is widespread and one that even pediatricians face constantly,” a Connecticut doctor ...

This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...

Spinal muscular atrophy type 1 is a genetic disorder that typically appears within the first six months of life, causing ...

Novartis also shared results from the open-label phase 3b STRENGTH study of OAV101 IT in SMA patients aged two to less than 18 years who had discontinued treatment with nusinersen or risdiplam, which ...

Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene ...