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 ...

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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 ...

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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. ...

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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. ...

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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. ...

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'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 ...

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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. ...

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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. ...

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'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 ...

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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 ...

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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 ...

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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. ...

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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 ...

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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 ...

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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 ...

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