Blog
RSS FeedPosted by Michael Wonder on 19 Jun 2020
Dicerna receives rare paediatric disease designation from U.S. FDA for nedosiran for the treatment of primary hyperoxaluria
18 June 2020 - Dicerna Pharmaceuticals today announced that the U.S. FDA has granted rare paediatric disease designation for nedosiran, ...
Posted by Michael Wonder on 12 Jun 2020
Orphan drug reimbursements create tough situation for South Korean public budget
12 June 2020 - A data analysis of real-world data found that the rise of orphan drugs in the Republic of ...
Posted by Michael Wonder on 11 Jun 2020
Rare disease therapy development and access remain top FDA priorities during COVID-19
11 June 2020 - The FDA’s work on behalf of people with rare diseases is more important than ever as these ...
Posted by Michael Wonder on 03 Jun 2020
Clarity Pharmaceuticals announces that the US FDA grants rare paediatric disease designation to Copper Cu 67 sartate for the treatment of neuroblastoma
3 June 2020 - Clarity Pharmaceuticals is pleased to announce that the U.S. FDA has granted rare paediatric disease designation to ...
Posted 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 ...