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 FDA restricts use of bluebird's Skysona gene therapy The FDA has added a warning label to bluebird bio's Skysona, after launching a probe into cases of blood cancer linked to the gene therapy.
News Jazz gets FDA okay for Chimerix brain cancer drug Jazz Pharmaceuticals has become the first drugmaker to get FDA approval for a treatment for diffuse midline glioma with H3 K27M mutations.
News Rumour mill says Novartis may want to buy Avidity Novartis has made an offer to acquire RNA therapeutic specialist Avidity Biosciences, according to an unconfirmed report.
News Sanofi grabs Chinese rights to Arrowhead rare disease drug Sanofi has paid $130 million upfront for Chinese rights to an Arrowhead Pharma drug for rare disease familial chylomicronaemia syndrome.
R&D Novel study designs for rare diseases Developing treatments for rare diseases poses many unique challenges that cannot be addressed by traditional study designs.
News Remepy's hybrid Parkinson's therapy poised for phase 3 Remepy's Hybridopa therapy for Parkinson's, which combines an app with levodopa therapy, has shown efficacy in a phase 2 pilot study.
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