PHARMAC funding increase "extremely disappointing"

11 May 2020 - Rare Disorders NZ is extremely disappointed with the announcement yesterday of a pre-budget boost to PHARMAC’s funding ...

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Recent PHARMAC funding decisions

22 April 2020 - You may have read about PHARMAC’S decision this week to scrap a lung cancer drug funding ...

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Gilead declines 'rare disease' status for experimental coronavirus drug

25 March 2020 - In a surprising turnabout, drugmaker Gilead Sciences asked the FDA on Wednesday to rescind orphan status ...

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Orphan Technologies receives rare paediatric disease designation from FDA for OT-58 to treat cystathionine B-synthase deficiency homocystinuria

26 March 2020 - Orphan Technologies today announced that OT-58 for the treatment of cystathionine beta synthase deficiency homocystinuria has ...

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Determinants of orphan drug prices in Germany

21 March 2020 - Legislation introduced in 2011 in Germany has instituted an early benefit assessment of newly licensed pharmaceuticals with ...

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Patients in the 'Fight for Our Lives' call on Parliament to STOP the PMPRB!

10 March 2020 - As the world comes together to fight coronavirus, Canadians with life-threatening rare and common diseases are ...

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Girl loses High Court battle over NICE review of BioMarin’s phenylketonuria drug

5 March 2020 - An 11-year-old girl with the rare disease phenylketonuria has lost a legal battle in the High ...

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Cystic fibrosis is the reason for denial of Irish man's re-entry visa by Australian Government, family says

26 February 2020 - A young Irish man with cystic fibrosis who calls Western Australia home has been denied entry ...

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First National Action Plan for rare diseases

26 February 2020 - The Australian Government will provide up to $3.3 million for activities to implement the first National Strategic ...

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Everyone wants to help, but still the red tape prevents Dylan Barker getting medicine

25 February 2020 - Dylan Barker lives with a rare genetic condition turning muscles, tendons and ligaments to bone, locking ...

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Rare Disease Day 2020: FDA continues important work on treatments for rare diseases

21 February 2020 - As leaders of the FDA, every day we see the unwavering commitment of our agency in working ...

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After a new version of a decades-old drug gets orphan status, the price suddenly skyrockets

12 February 2020 - For years, hospitals and clinics have used an injectable medicine called dehydrated alcohol to treat such ...

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SMC approves first two ‘ultra-orphan’ pathway meds

11 February 2020 - The Scottish Medicines Consortium has released its February guidance, advising six new medicines to NHS Scotland. ...

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SMC - February 2020 decisions

10 February 2020 - The Scottish Medicines Consortium, which advises on newly licensed medicines for use by NHSScotland, has today ...

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ProQR receives rare paediatric disease designation from FDA for QR-421a

30 January 2020 - Designation is for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 ...

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