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.
Concern about the safety of gene therapies has been thrust into the spotlight again with the news that two patients treated with Novartis' spinal muscular atrophy (SMA) treatment Zolgensma
Spinal muscular atrophy (SMA) is a devastating, genetic neuromuscular disease caused by a lack of a functional SMN1 gene that results in the progressive and irreversible loss of motor neuro
Spinal muscular atrophy (SMA) therapy Spinraza has been a key earner for Biogen in recent years, but with sales now heading into reverse, the company is looking to extend its franchise.
Astellas has added another gene therapy to its pipeline, taking an option on a clinical-stage candidate for a rare genetic form of dementia from UK biotech AviadoBio for around $50 million.