Blog
RSS FeedPosted by Michael Wonder on 11 May 2020
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 ...
Posted by Michael Wonder on 23 Apr 2020
Recent PHARMAC funding decisions
22 April 2020 - You may have read about PHARMAC’S decision this week to scrap a lung cancer drug funding ...
Posted by Michael Wonder on 26 Mar 2020
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 ...
Posted by Michael Wonder on 26 Mar 2020
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 ...
Posted by Michael Wonder on 23 Mar 2020
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 ...
Posted by Michael Wonder on 11 Mar 2020
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 ...
Posted by Michael Wonder on 06 Mar 2020
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 ...
Posted by Michael Wonder on 27 Feb 2020
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 ...
Posted by Michael Wonder on 27 Feb 2020
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 ...
Posted by Michael Wonder on 25 Feb 2020
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 ...
Posted by Michael Wonder on 23 Feb 2020
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 ...
Posted by Michael Wonder on 16 Feb 2020
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 ...
Posted by Michael Wonder on 11 Feb 2020
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. ...
Posted by Michael Wonder on 11 Feb 2020
SMC - February 2020 decisions
10 February 2020 - The Scottish Medicines Consortium, which advises on newly licensed medicines for use by NHSScotland, has today ...
Posted by Michael Wonder on 30 Jan 2020
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 ...