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

A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...

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

Novartis AG (NYSE:NVS) announced safety and efficacy results from the Phase 3 program for investigational intrathecal ...

In this trial, the efficacy and safety of the therapy were evaluated against a sham control in treatment-naïve SMA Type 2 ...

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

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

Baclofen 50mcg/mL, 500mcg/mL, 2000mcg/mL; for intrathecal inj; preservative-free. <4yrs: not established. ≥4yrs: Give test dose 1 st by intrathecal inj via spinal catheter or lumbar puncture ...

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

High-dose methotrexate (HD-MTX) with the modified Bonn protocol including intrathecal therapy is effective for patients less than 65 years old with primary central nervous system lymphoma (PCNSL), ...