Revelations of data manipulation might not be fatal, but they will hurt the chief exec’s attempted rebranding of Novartis as a more ethical pharma company.
Competition is looming from Novartis and Roche but the latest results from Spinraza show that Biogen still has a strong case to make for the SMA therapy.
Worries over toxicity with Pfizer's PF-06939926 halts trial dosing and could leave the way clear for its DMD rival Sarepta.
Despite concerns about a missing patient, results from a tiny trial in epidermolysis bullosa boosted Krystal Biotech’s stock by 41%.
The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.
Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
Three-year data from Biomarin’s haemophilia A gene therapy fail to quash concerns about durability, but the company insists that the results are good enough to file on.
The US FDA granted seven approvals in two days last week, including an eagerly awaited thumbs-up for Novartis’s gene therapy Zolgensma.
Expectations are low for a three-year update on the group’s haemophilia A gene therapy, and Biomarin’s rivals could capitalise on any slip-up.