Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 22 Jul 2019
Vertex submits new drug application to the U.S. FDA for triple combination regimen of VX-445 (elexacaftor), tezacaftor and ivacaftor in cystic fibrosis
22 July 2019 - Application supported by positive results from two global Phase 3 studies in people with CF ages 12 ...
Posted by Michael Wonder on 17 Jul 2019
Perth boy battles cystic fibrosis, with a life extending drug frustratingly out of reach
15 July 2019 - In many ways Connor Barrett is just like any other kid - he loves Peppa Pig ...
Posted by Michael Wonder on 16 Jul 2019
Irish cystic fibrosis family face €12,400 bill after deportation victory in Australia
15 July 2019 - An Irish family threatened with deportation from Australia because their son has cystic fibrosis have been ...
Posted by Michael Wonder on 11 Jul 2019
Reforming the Orphan Drug Act for the 21st century
11 July 2019 - The pharmaceutical market has undergone radical changes, including markedly increased prices for rare-disease drugs. ...
Posted by Michael Wonder on 05 Jul 2019
Government breaks promises to Pompe disease sufferers
5 July 2019 - The New Zealand Pompe Network recently presented a petition at Parliament asking that the House of ...
Posted by Michael Wonder on 04 Jul 2019
Ultomiris (ravulizumab) receives marketing authorisation from European Commission for adults with paroxysmal nocturnal hemoglobinuria
3 July 2019 - Ultomiris is the first approved, long-acting complement inhibitor for PNH, administered every other month, reducing the ...
Posted by Michael Wonder on 03 Jul 2019
New hope for patients with rare diseases with the launching of a new fund
2 July 2019 - Singaporeans with three forms of rare diseases now have access to financial aid to help with ...
Posted by Michael Wonder on 20 Jun 2019
U.S. FDA accepts supplemental biologics license application for Ultomiris (ravulizumab-cwvz) under priority review for the treatment of atypical haemolytic uremic syndrome
20 June 2019 - FDA sets target action date of 19 October 2019. ...
Posted by Michael Wonder on 19 Jun 2019
Abeona Therapeutics receives FDA fast track designation for ABO-202 AAV9 gene therapy in CLN1 disease
18 June 2019 - Abeona Therapeutics today announced that the U.S. Food and FDA has granted fast track designation to its ...
Posted by Michael Wonder on 18 Jun 2019
Buyers’ Club wants government support on Orkambi imports
17 June 2019 - Proposes three point plan to work round impasse. ...
Posted by Michael Wonder on 11 Jun 2019
Jacobus prices its rare disease drug at half of what Catalyst charges, but will doctors prescribe it?
10 June 2019 - After weeks of anticipation, Jacobus Pharmaceutical, a small, family-run drug maker, has priced its rare disease ...
Posted by Michael Wonder on 11 Jun 2019
MPs to debate cystic fibrosis drug Orkambi in Parliament
10 June 2019 - A debate is due to take place in Westminster about a drug for people living with ...
Posted by Michael Wonder on 11 Jun 2019
Orkambi row: government now considering Crown Use licensing
11 June 2019 - MPs call for radical solutions as deadlock remains. ...
Posted by Michael Wonder on 06 Jun 2019
Enzyvant announces FDA acceptance of biologics license application and priority review status for RVT-802, a novel investigational tissue-based regenerative therapy for paediatric congenital athymia
5 June 2019 - RVT-802, a one-time therapy, leverages Enzyvant’s T cell generation platform designed to treat profound immunodeficiencies. ...