Perth boy battles cystic fibrosis, with a life extending drug frustratingly out of reach

15 July 2019 - In many ways Connor Barrett is just like any other kid - he loves Peppa Pig ...

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Irish cystic fibrosis family face €12,400 bill after deportation victory in Australia

15 July 2019 - An Irish family threatened with deportation from Australia because their son has cystic fibrosis have been ...

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Reforming the Orphan Drug Act for the 21st century

11 July 2019 - The pharmaceutical market has undergone radical changes, including markedly increased prices for rare-disease drugs.  ...

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Government breaks promises to Pompe disease sufferers

5 July 2019 - The New Zealand Pompe Network recently presented a petition at Parliament asking that the House of ...

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Ultomiris (ravulizumab) receives marketing authorisation from European Commission for adults with paroxysmal nocturnal hemoglobinuria

3 July 2019 - Ultomiris is the first approved, long-acting complement inhibitor for PNH, administered every other month, reducing the ...

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New hope for patients with rare diseases with the launching of a new fund

2 July 2019 - Singaporeans with three forms of rare diseases now have access to financial aid to help with ...

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U.S. FDA accepts supplemental biologics license application for Ultomiris (ravulizumab-cwvz) under priority review for the treatment of atypical haemolytic uremic syndrome

20 June 2019 - FDA sets target action date of 19 October 2019. ...

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Abeona Therapeutics receives FDA fast track designation for ABO-202 AAV9 gene therapy in CLN1 disease

18 June 2019 - Abeona Therapeutics today announced that the U.S. Food and FDA has granted fast track designation to its ...

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Buyers’ Club wants government support on Orkambi imports

17 June 2019 - Proposes three point plan to work round impasse. ...

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Jacobus prices its rare disease drug at half of what Catalyst charges, but will doctors prescribe it?

10 June 2019 - After weeks of anticipation, Jacobus Pharmaceutical, a small, family-run drug maker, has priced its rare disease ...

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MPs to debate cystic fibrosis drug Orkambi in Parliament

10 June 2019 - A debate is due to take place in Westminster about a drug for people living with ...

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Orkambi row: government now considering Crown Use licensing

11 June 2019 - MPs call for radical solutions as deadlock remains. ...

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Enzyvant announces FDA acceptance of biologics license application and priority review status for RVT-802, a novel investigational tissue-based regenerative therapy for paediatric congenital athymia

5 June 2019 - RVT-802, a one-time therapy, leverages Enzyvant’s T cell generation platform designed to treat profound immunodeficiencies. ...

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Families create buyers club for cut-price cystic fibrosis drug

5 June 2019 - Stalemate in NHS talks over Orkambi leads parents to buy generic version from Argentina. ...

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US FDA review for Scenesse extended by three months

3 June 2019 - The FDA sets new goal date of 6 October 2019 to complete review and issue risk-benefit decision. ...

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