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
The controversy over rising drug prices in the US has been reignited after it emerged that the yearly cost of the life-sustaining rare disease drug Firdapse has been hiked from free to more