Swiss biotech Chord Therapeutics has come out of stealth mode with a $16 million first-round financing that will be used to progress a repurposed drug for rare disease neuromyelitis optica
Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.<
Analysts have said the FDA’s request for a new trial will delay Novartis’ intrathecal formulation of its spinal muscular atrophy (SMA) gene therapy in older patients until 2023, potentially
Patients with the rare disease familial chylomicronemia syndrome (FCS) will be able to access treatment with Akcea’s Waylivra via the NHS, after a U-turn by NICE.
The FDA has started a speedy review of Regeneron’s ANGPTL3-targeting antibody evinacumab for a rare, inherited disorder that dramatically raises the risk of heart disease.<
The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase alfa, setting up a possible approval by 27
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