Novartis unveils positive study data on Zolgensma gene therapy
Novartis has presented new study data on Zolgensma, its gene therapy treating spinal muscular atrophy (SMA). The data once again underscored the efficacy of this single-dose gene therapy in patients with SMA, according to a press release issued on Monday.
The results of the SMART study added to what is known about the use of Zolgensma in patients who are older (1.5 – 9.1 years) and suffer more severely than the children treated in previous clinical trials.
In almost all cases, the motoric milestones of treated patients were maintained or even improved after 52 weeks. Many of these patients had switched therapies, having previously been treated with a different disease-modifying agent.
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The SMART study is reportedly the first open-label clinical trial of Zolgensma to include previously treated patients. Novartis announced that it will present the data at the Muscular Dystrophy Association (MDA) conference, which is taking place in the US until March 6.
Adapted from German by DeepL/dkk/amav
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