News UCB drug for rare disease TK2d boosts survival UCB's experimental medicine for TK2d, currently under regulatory review, offers hope to patients with the devastating rare disease.
News Oculis takes second eye disease therapy into pivotal trials Oculis is poised to start registration trials for privosegtor, its drug candidate for neuro-ophthalmic diseases, in the wake of positive phase 2 data.
News Acadia, Harmony record late-stage rare disease failures Biotechs Acadia Pharma and Harmony Biosciences both suffered setbacks in phase 3 trials of drugs for rare diseases, in one case ending the programme.
News Stealth's patience rewarded as FDA approves Barth drug After a long haul, Stealth BioTherapeutics has finally claimed FDA approval for Forzinity, the first treatment for ultra-rare disease Barth syndrome.
News Regeneron drug on track to be second FOP treatment There is only one approved drug for ultra-rare disease FOP on the market, but Regeneron is hoping to add a second.
News Servier bets $450m on Fragile X syndrome drug Servier has pushed its emerging neurology pipeline into the clinical trials stage by licensing a drug for Fragile X syndrome from the UK's Kaerus.
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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