Roche is to buy gene therapy firm Spark Therapeutics for $4.8 billion, adding an already-approved treatment for inherited blindness to its portfolio, and boosting its pipeline with a series
Catalyst Pharmaceuticals has defended hiking the price of the rare disease drug Firdapse (amifampridine) from virtually nothing to more than $300,000 per year following criticism from Senat
UK-based biotech ReNeuron has announced encouraging results from an early stage trial of its cell therapy for the rare blindness-causing disease, retinitis pigmentosa (RP).
UK health technology startup Mendelian has received a £500,000 grant from a government agency to build products that will help GPs identify patients with rare or hard to diagnose conditions
The US regulator has approved Novo Nordisk’s long-acting therapy for haemophilia A, Esperoct, as the Danish pharma seeks to maintain its presence in an increasingly competitive sector of th
