Shares in Scholar Rock have gone into overdrive after the biotech reported positive phase 3 results with apitegromab in patients with spinal muscular atrophy (SMA), setting up regulatory fi
Novartis has doubled down on its gene therapy collaboration with Voyager Therapeutics, stumping up another $1.2 billion-plus in potential milestone payments and directing
Novartis' programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company.
