The US Zolgensma Review: Full Speed Ahead Despite Dose, Stability, Method Uncertainties
Normally, toddlers with infantile-onset spinal muscular atrophy would be in bed on ventilators. But most of these were sitting up on their own. Two were even standing and walking. The question was how to get the manufacturing process for the gene therapy that worked this magic ready for prime time.
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Keeping Track: Novartis Scores Big Ahead Of US Memorial Day With Approvals For Gene Therapy Zolgensma, Oncologic Piqray
The Swiss major has made a larger-than-expected bolt-on buy, using a large chunk of its $13bn windfall from GSK to get hold of a potential cure for spinal muscular atrophy and an additional gene therapy platform, as well as manufacturing capabilities.
Just as they did during the heparin crisis a decade ago, pharmaceutical companies must coordinate on coronavirus, Rx-360 CEO says.