Blog
RSS FeedPosted by Michael Wonder on 24 Oct 2019
NHSE signs ‘definitive’ agreement for Vertex CF drugs
24 October 2019 - NHS England has announced a definitive agreement with Vertex Pharmaceuticals to make all three of their ...
Posted by Michael Wonder on 08 Oct 2019
FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases
8 October 2019 - The U.S. FDA today announced that it has awarded 12 new clinical trial research grants totaling ...
Posted by Michael Wonder on 06 Oct 2019
Mum 'hopeful' after baby Kaan Hunter starts $250k a year medication for rare bone disease
5 October 2019 - A mum whose baby was the first Kiwi to be diagnosed with a rare bone disease ...
Posted by Michael Wonder on 17 Sep 2019
News NHS England explores funding options for cystic fibrosis drug
17 September 2019 - NHS England is “committed to exploring and developing alternative routes” to enable people with cystic fibrosis ...
Posted by Michael Wonder on 13 Sep 2019
Vertex announces new access agreement with Scottish Government for Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor)
12 September 2019 - Eligible patients in Scotland will immediately have access to Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) in combination with ...
Posted by Michael Wonder on 12 Sep 2019
Batten disease treatment: parents win battle ahead of court date
11 September 2019 - Families of children with a rare degenerative disease have won their fight for funding for NHS ...
Posted by Michael Wonder on 11 Sep 2019
Children with rare inherited condition to benefit from drug through managed access agreement
11 September 2019 - It is estimated that in the UK there are around 30 to 50 children living with the ...
Posted by Michael Wonder on 03 Sep 2019
The FDA and Sarepta: a window into the real world of drug regulation
3 September 2019 - It is hard to discern the true state of drug regulation from the outside, but two ...
Posted by Michael Wonder on 28 Aug 2019
European Commission approves Soliris (eculizumab) for the treatment of adults with neuromyelitis optica spectrum disorder
27 August 2019 - 98% of adult anti-aquaporin-4 antibody-positive patients treated with Soliris were relapse free compared to 63% receiving placebo ...
Posted by Michael Wonder on 20 Aug 2019
FDA accepts new drug application for VX-445 (elexacaftor), tezacaftor and ivacaftor combination treatment
20 August 2019 - FDA grants priority review of the application and sets a PDUFA target action date of 19 ...
Posted by Michael Wonder on 13 Aug 2019
Canadian drug price regulator may be flexible on rare diseases
14 August 2019 - Canada’s patented drug price regulator, set to gain new powers next year, may be “more forgiving” in ...
Posted by Michael Wonder on 31 Jul 2019
Medicines access for cancer must also be Fair for Rare
30 July 2019 - Rare Disorders NZ welcomes the National Party’s recent announcement on cancer care but calls on both ...
Posted by Michael Wonder on 24 Jul 2019
Aeglea BioTherapeutics receives FDA breakthrough therapy designation for pegzilarginase for treatment of arginase 1 deficiency
24 July 2019 - Designation follows recently reported ARG1-D Phase 1/2 data demonstrating clinical response in patients rreated with pegzilarginase. ...
Posted by Michael Wonder on 24 Jul 2019
Patients, backed by ‘dark money’ group, get ready to debate Duchenne drug prices. It will not be boring.
24 July 2019 - Meetings of the Institute for Clinical and Economic Review don’t usually get one’s heart pumping. ...
Posted by Michael Wonder on 23 Jul 2019
Mum needs $100,000 to treat baby son with infantile hypophosphatasia
23 July 2019 - A baby boy will only live for a few months without a $100,000 medication that is ...