Blog
RSS FeedPosted by Michael Wonder on 13 Nov 2019
Regulators’ advice can make a difference for faster patient access to highly innovative therapies
12 November 2019 - Early dialogue and frequent interactions between medicine developers, regulators, health technology assessment bodies and patients can play ...
Posted by Michael Wonder on 06 Nov 2019
Novartis says delayed telling FDA of Zolgensma concern due to 'mistake'
2 November 2019 - Novartis scientists learnt months ago about safety concerns surrounding its gene therapy Zolgensma but delayed telling the ...
Posted by Michael Wonder on 06 Nov 2019
One shot drug to end Sicilian curse comes at $1.8 million cost
5 November 2019 - A new targeted gene therapy shows the promise and pitfalls of medical advances. ...
Posted by Michael Wonder on 05 Nov 2019
Talking with Tama, a sick toddler who can't access the drug he needs
5 November 2019 - "Hi!" a bright young voice says down the phone. ...
Posted by Michael Wonder on 03 Nov 2019
Tama is deteriorating fast. But the drug that can help him is too expensive.
31 October 2019 - Tama Hubbard has good days and bad days, just like any other 3-year-old child. ...
Posted by Michael Wonder on 01 Nov 2019
'Why won’t they help him?': 'Broken' mother pleads for PHARMAC funding for toddler's rare disorder
1 November 2019 - The mother of a young boy is pleading with PHARMAC to fund a drug that could ...
Posted by Michael Wonder on 22 Oct 2019
Moderna receives FDA fast track designation for propionic acidemia program (mRNA-3927)
22 October 2019 - mRNA-3927 is Moderna’s second rare disease program to receive fast track designation. ...
Posted by Michael Wonder on 17 Oct 2019
Canadian breakthrough that became the world's most expensive drug, then vanished, gets second chance
17 October 2019 - CBC story about how pharmaceutical industry abandoned gene therapy Glybera sparks NRC effort to re-invent it. ...
Posted by Michael Wonder on 10 Oct 2019
'I'm not ready to bring her back' - NZ not safe for three-year-old with motor neuron disease, mum says
10 October 2019 - Three-year-old New Zealander Charlotte Bond is showing signs she may walk again on her own with ...
Posted by Michael Wonder on 28 Sep 2019
AveXis rules out EU compassionate use of SMA gene therapy, says Biogen option is available
27 September 2019 - Novartis’ AveXis unit has given its reasons for refusing to supply its spinal muscular atrophy gene ...
Posted by Michael Wonder on 24 Sep 2019
Novartis unit says FDA wasn’t told of data manipulation sooner because it was ‘highly complex’
25 September 2019 - If you were wondering why Novartis waited more than three months to notify the Food and ...
Posted by Michael Wonder on 24 Sep 2019
FDA grants paediatric disease designation for Mateon's OT101
23 September 2019 - For treatment of diffuse intrinsic pontine glioma, a difficult to treat form of childhood brain tumour. ...
Posted by Michael Wonder on 18 Sep 2019
Baby Pia: almost 1 million Belgians pay for life-saving drug
18 September 2019 - More than 950,000 Belgians have responded to a couple seeking €1.9m ($2m; £1.7m) to cover the ...
Posted by Michael Wonder on 18 Sep 2019
Three-year-old Matilda's ability to walk is giving hope to those with spinal muscular atrophy
18 September 2019 - The disease, which has been described as motor neurone disease for children, is the leading cause of ...
Posted by Michael Wonder on 15 Sep 2019
The startup that manipulated data to get a miracle drug to market
14 September 2019 - AveXis had a revolutionary gene therapy for babies with a deadly disease, and sold to Novartis for ...