Blog
RSS FeedPosted by Michael Wonder on 08 Mar 2021
World's most expensive drug given the green light in Scotland
8 March 2021 - A medicine known as the most expensive in the world has been approved to treat a rare ...
Posted by Michael Wonder on 08 Mar 2021
SMC - March 2021 decisions
8 March 2021 - Medicines which can be used to treat a rare genetic disease in children and some cancer ...
Posted by Michael Wonder on 08 Mar 2021
NICE approves life changing gene therapy for treating spinal muscular atrophy
8 March 2021 - A new and potentially curative one-off gene therapy for babies with the rare genetic disorder spinal muscular ...
Posted by Michael Wonder on 01 Mar 2021
Nusinersen in spinal muscular atrophy: indication of considerable added benefit in children with an early onset of the disease
1 March 2021 - The treatment delays permanent ventilation of the children and extends life. In addition, the children develop more ...
Posted by Michael Wonder on 17 Feb 2021
bluebird bio ‘baffled’ after NICE rejects beta thalassaemia gene therapy
16 February 2021 - It’s back to the drawing board for bluebird bio and its discussions with NICE, which has ...
Posted by Michael Wonder on 28 Jan 2021
Alberta announces funding for pricey gene therapy drug Zolgensma
Alberta Health Minister Tyler Shandro announced funding Wednesday for the gene therapy Zolgensma, which is used to treat toddlers with ...
Posted by Michael Wonder on 21 Dec 2020
Newly approved treatment could save a young child in Edmonton with a rare disease. It costs 2.8 million dollars per dose.
19 December 2020 - Reign Johnston is a happy baby who enjoys painting as much as playing with toys. His green ...
Posted by Michael Wonder on 18 Dec 2020
Recommendations made by the PBAC - November 2020
18 December 2020 - The recommendations from the November 2020 PBAC meeting are now available. ...
Posted by Michael Wonder on 15 Nov 2020
Life-saving spinal muscular atrophy medicine to be made available to Australian babies
15 November 2020 - Access to a life-saving and life-changing medicine will soon be expanded for Australian children and infants with ...
Posted by Michael Wonder on 21 Oct 2020
Canadian baby with spinal muscular atrophy to get ‘world’s most expensive drug’
20 October 2020 - A little over a year ago, the idea of raising US$2.125 million to receive the “world’s ...
Posted by Michael Wonder on 16 Sep 2020
Human Rights Commission grants case against PHARMAC for failing to fund rare muscular disorder drug
16 September 2020 - Newshub can reveal a ground-breaking Human Rights Commission case has been taken against Pharmac for failing ...
Posted by Michael Wonder on 14 Sep 2020
Stranded mum with dying daughter makes desperate plea to Jacinda Ardern
13 September 2020 - A distraught mum stuck in New Zealand with her dying daughter Stella amid the COVID pandemic ...
Posted by Michael Wonder on 12 Sep 2020
Kiwi mum pleads with PHARMAC to fund life-changing drug Spinraza for her two children
11 September 2020 - The mother of two young boys with a rare disease is begging PHARMAC to fund a ...
Posted by Michael Wonder on 12 Sep 2020
NICE widens its Zolgensma appraisal due to European Marketing Authorisation
11 September 2020 - England’s NICE is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy, according ...
Posted by Michael Wonder on 15 Aug 2020
Family battles PHARMAC and eyes Australian move to get 'miracle drug'
15 August 2020 - When Lani McLeod found out her toddler would never walk, she was gutted. ...