Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA: Basel Friday, March 21, 2025, 11:00 Hrs [IST] N ...

Novartis (NVS) announced that oral Fabhalta, iptacopan, has received U.S. Food and Drug Administration approval for the ...

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

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

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.

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 has reported positive efficacy and safety outcomes from its Phase III clinical programme for OAV101 IT (onasemnogene ...

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

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

Swiss pharma major Novartis (NOVN: VX) has announced positive results from its Phase III STEER study, evaluating the efficacy ...