Rare diseases

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BridgeBio two for two as rare disease drug aces ph3 trial

BridgeBio's week just went from great to superb, as it reports a second positive topline result in a pivotal trial of a late-stage pipeline drug.

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Merck looking to rare disease deals to build health momentum

Germany's Merck KGaA is looking at making further acquisitions to boost its business, with a focus on life sciences and rare diseases.

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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.

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Black and white close up image of an eye

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.

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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.

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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.

BridgeBio two for two as rare disease drug aces ph3 trial

Merck looking to rare disease deals to build health momentum

UCB drug for rare disease TK2d boosts survival

Oculis takes second eye disease therapy into pivotal trials

Acadia, Harmony record late-stage rare disease failures

Stealth's patience rewarded as FDA approves Barth drug

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Rare diseases

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