LogicBio Therapeutics receives rare paediatric disease designation for LB-001 for the treatment of methylmalonic acidaemia

16 July 2019 -  LogicBio Therapeutics today announced the U.S. FDA has granted rare paediatric disease designation to LB-001, a ...

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Dicerna receives breakthrough therapy designation for DCR-PHXC for treatment of primary hyperoxaluria type 1

15 July 2019 - FDA recognises primary hyperoxaluria Types 2 and 3 as meeting criteria for a serious or life-threatening disease ...

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'Amazing' gene-silencing drugs reach NHS

9 July 2019 - A new form of medicine called "gene-silencing" has been approved for use by the NHS in ...

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NICE Onpattro approval puts Alnylam back in hATTR running

 9 July 2019 - NICE has recommended the use of Alynylam’s Onpattro (patisiran), after issuing a preliminary ‘no’, back in ...

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Report calls for European funding reform for gene therapies

8 July 2019 - New payment models, dedicated investment, and better health technology assessment are among a raft of changes ...

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Life-changing drug Spinraza still not covered for many Canadians with spinal muscular atrophy

4 July 2019 - For several hundred Canadians with spinal muscular atrophy, Spinraza is a drug that means the difference ...

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Spinraza access agreement extended

3 July 2019 - More children with the rare genetic disorder spinal muscular atrophy can now be treated with Spinraza after ...

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Some insurers are pushing back on the high price for a Novartis gene therapy

3 July 2019 - Barely a month after Novartis won U.S. regulatory approval for its gene therapy — the most ...

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These two brothers are living healthier lives, thanks to gene therapy

3 July 2019 - It has taken decades of work and billions of dollars, but Australia is now entering an ...

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Innovation prizes to support cell and gene therapy

2 July 2019 - Personalised cell and gene therapy represent a fundamental therapeutic revolution.  ...

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Alnylam submits marketing authorisation application to the EMA for givosiran for the treatment of acute hepatic porphyria

1 July 2019 - Patients receiving givosiran in pivotal Phase 3 ENVISION study had a 74% mean reduction in annualised rate ...

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Krystal Biotech receives regenerative medicine advanced therapy designation from FDA for KB103

24 June 2019 - RMAT designation granted to KB103 based on positive interim data from GEM-1 and GEM-2 study. ...

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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 ...

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Zolgensma: a remarkable new treatment, an ICER analysis, and a poorly justified price

18 June 2019 - Defects in both sets of alleles in the coding region for the survival motor neuron 1 ...

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New cost-effectiveness methods to determine value-based prices for potential cures: what are the options?

12 June 2019 - Evaluating different approaches to assessing the clinical effectiveness and value of potential cures will be essential to ...

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