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

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

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

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On Wednesday, Novartis gave a more detailed look at long-awaited clinical data that the company believes will help secure a ...

The FDA wants to end bulk production of copycat versions of weight loss drugs; telehealth companies and compounding ...

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

Novartis released two Phase 3 datasets on Wednesday that show a new version of its gene therapy Zolgensma led to motor ...

This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal ...

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

Novartis' drug segment is poised for steady ... (hidradenitis suppurativa in 2023), spinal muscular atrophy drug Zolgensma (intrathecal data in 2024), and cardiovascular drug Leqvio (outcomes ...