News NHS cleared to use AZ's Fasenra for rare autoimmune disease NICE backs NHS use of an AstraZeneca drug for one rare disease, but says it can't recommend a BioMarin therapy for another.
News Final NICE rejection for Biomarin's childhood rare disease d... After a year of negotiations NICE has confirmed that it is still unable to recommend Biomarin’s Batten disease drug, saying that the company is unable to address concerns about long-term ef
News Agios shares slide as it abandons sickle cell candidate Agios Pharma has said it will halt clinical development of tebapivat, a PK activator that had reached phase 2 testing in sickle cell disease.
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