To have any impact, patient engagement needs to start with the drug development pathway, not at the point of licensing. That’s especially true in the rare disease space, where the unme
The FDA has granted a fast review for Alexion Pharmaceuticals’ filing for its Soliris, as a treatment for a rare neurological disease that leads to worsening disability, blindness, paralysi
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).