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
Phase 2 results with Wave Life Sciences' Duchenne muscular dystrophy candidate WVE-N531 pushed the company's share price to its highest level in a year and could lead to discussions with re
Sanofi has bolstered the case for its oral BTK inhibitor tolebrutinib as a treatment for multiple sclerosis (MS), due to be filed for approval in the latter half of this year, with new data
Data from a phase 2 trial of Roche's oral BTK inhibitor fenebrutinib have shown "near-complete suppression" of disease activity and disability progression in relapsing multiple sclerosis (M
Sanofi has said it plans to start discussions about filing its oral BTK inhibitor tolebrutinib for multiple sclerosis, even though the drug only achieved its primary objective in one of thr
Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy, the biotech has closed a public offering that raised an imp
Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share pric
Investigator sites are essential for successful execution of clinical trials – ensuring studies are conducted ethically, safely, and in compliance with regulatory standards.