Blog
RSS FeedPosted by Michael Wonder on 27 May 2016
Muscular dystrophy day at the FDA
25 May 2016 - The agency again delays a decision on a therapy for a deadly disease. ...
Posted by Michael Wonder on 19 May 2016
Sydney mother calls for cystic fibrosis drug Orkambi to be accessible for daughter
20 May 2016 - West Pymble six-year-olds Charli and Jasmine are twins, but their future lives look very different. ...
Posted by Michael Wonder on 16 May 2016
PHARMAC’s new approach for people with rare disorders
16 May 2016 - A new approach PHARMAC has used for funding medicines for rare disorders is gaining real traction, says ...
Posted by Michael Wonder on 12 May 2016
Unfair if rare: should the PBS change the way it lists cancer drugs?
12 May 2016 - The PBS spends over A$9 billion a year subsidising a wide range of drugs to ensure affordability ...
Posted by Michael Wonder on 12 May 2016
Decision to list bedaquiline (Sirturo) and siltuximab (Sylvant) for rare disorders
12 May 2016 - PHARMAC is pleased to announce the approval of an agreement with Janssen-Cilag for the listing of bedaquiline ...
Posted by Michael Wonder on 04 May 2016
BioMarin announces EMA grants accelerated assessment for cerliponase alfa, experimental treatment for a form of Batten disease
3 May 2016 - BioMarin today announced that the EMA has granted BioMarin's request for accelerated assessment for the planned cerliponase ...
Posted by Michael Wonder on 04 May 2016
Pharmaceutical Benefits Scheme – new and amended listings
3 May 2016 - This budget measure will make new and amended medicines available on the Pharmaceutical Benefits Scheme (PBS) and ...
Posted by Michael Wonder on 02 May 2016
Cystic fibrosis patients campaign for drug Orkambi to be listed on Pharmaceutical Benefits Scheme
2 May 2016 - Without subsidy, it costs about US $260,000 each year. ...
Posted by Michael Wonder on 26 Apr 2016
How can a price be put on my daughter's life?'
27 April 2016 - A Sunshine Coast mother has failed in her attempt to have the Federal Government list on ...
Posted by Michael Wonder on 25 Apr 2016
The FDA vs. Austin Leclaire
25 April 2016 - The agency sits on a new treatment for a deadly muscular disease. ...
Posted by Michael Wonder on 23 Apr 2016
Breakthrough cystic fibrosis drug rejected by Australian authorities
22 April 2016 - A break-through drug heralded to dramatically reduce the impacts of cystic fibrosis will not be added to ...
Posted by Michael Wonder on 22 Apr 2016
Record of stakeholder meeting - eculizumab (Soliris) for atypical haemolytic uraemic syndrome (February 2016)
22 April 2016 - A clinical advice meeting was held on 12 February 2016 to discuss the place of eculizumab (Soliris) ...
Posted by Michael Wonder on 19 Apr 2016
Survey of healthcare experiences of Australian adults living with rare diseases
19 April 2016 - Few studies have examined whether the healthcare needs of people living with rare diseases are being met. ...
Posted by Michael Wonder on 15 Apr 2016
NICE recommends ataluren for treating Duchenne muscular dystrophy caused by a nonsense mutation
15 April 2016 - NICE has today recommended ataluren (Translarna, PTC Therapeutics) for treating children aged 5 and over with Duchenne ...
Posted by Michael Wonder on 12 Apr 2016
Proposal to list bedaquiline fumarate (Sirturo) and siltuximab (Sylvant) for rare disorders
12 April 2016 - PHARMAC is seeking feedback on a proposal to list bedaquiline fumarate (Sirturo) and siltuximab (Sylvant) resulting from ...