Rare diseases

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Is pharma’s interest in orphan drugs waning?

For many years, orphan drugs for rare diseases have been a draw for pharma companies, offering high prices, swift regulatory reviews, and often extended marketing exclusiv

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With $75m cash injection, SynOx plans ph3 rare tumour trial

Ireland’s SynOx Therapeutics has completed a second-round financing, raising $75 million, as it prepares to take its drug for tenosynovial giant cell tumour (TGCT) into ph

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Ubie AI aims to improve diagnosis of rare disease Cushing’s

People with the rare disease Cushing’s syndrome can sometimes take years to get a diagnosis, but a new project hopes to shorten that timeline with the help of artificial i

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Sarah was born with ADA-SCID but was fortunate to access gene therapy via a clinical trial

NHS trust bids to take license for abandoned gene therapy

An NHS trust has taken the pioneering step of applying to hold the marketing authorisation for a gene therapy for a rare disease that was abandoned by a biotech developer

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EMA starts review of Rocket’s Fanconi anaemia gene therapy

Patients in the EU with Fanconi anaemia (FA) could soon have the first gene therapy option for the disorder, as the EMA starts a review of Rocket Pharma’s RP-L102 candidat

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FDA clears AZ’s new PNH therapy Voydeya

AstraZeneca’s Voydeya has become the first oral Factor D inhibitor to be approved by the FDA as a treatment for the rare disease paroxysmal nocturnal haemoglobinuria (PNH)

Is pharma’s interest in orphan drugs waning?

With $75m cash injection, SynOx plans ph3 rare tumour trial

Ubie AI aims to improve diagnosis of rare disease Cushing’s

NHS trust bids to take license for abandoned gene therapy

EMA starts review of Rocket’s Fanconi anaemia gene therapy

FDA clears AZ’s new PNH therapy Voydeya

AZ claims another win for Imfinzi in frontline liver cancer

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Rare diseases

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