BioMarin Pharmaceutical has put on a stage musical to raise awareness about haemophilia, and determine whether creative expression helps patients, as it develops an experimental cure for th
Novartis says it thinks its one-off gene therapy for spinal muscular atrophy (SMA) could deliver value for money even if its price was set upwards of $4 million – which would set a record f
Alexion has announced a collaboration with Dicerna Pharmaceuticals to discover and develop RNA interference therapies for diseases caused by problems in the complement system.
