Ireland an outlier without access to new cell and gene therapy, report says

27 May 2021 - Upfront costs high but treatments may cut long-term expense and boost patient results. ...

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Colchester baby's parents feel 'abandoned' over £1.7 million drug

25 May 2021 - A baby with a fatal genetic condition has been "abandoned" in a "race against time" for ...

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European Medicines Agency grants BioMarin's request for accelerated assessment of valoctocogene roxaparvovec for treatment of severe haemophilia A

24 May 2021 - MAA resubmission on track for June 2021. ...

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First gene therapy to treat children with rare inherited neurological disease

21 May 2021 - The EMA has recommended granting a marketing authorisation in the European Union for the gene therapy ...

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FDA: flexibility okay for gaps in gene therapy trials

18 May 2021 - The US FDA is not amenable to changing study outcome measures or sample sizes for gene therapy ...

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BridgeBio Pharma receives FDA fast track designation for investigational gene therapy for congenital adrenal hyperplasia

14 May 2021 - BridgeBio Pharma today announced that the U.S. FDA granted fast track designation to BBP-631, an investigational adeno-associated ...

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Dutch advisory group says Zolgensma shouldn’t be covered unless Novartis halves price

10 May 2021 - In a challenge to Novartis, a Dutch advisory body has recommended that the government not cover ...

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World’s most expensive drug may not be covered by Dutch insurance

7 May 2021 - A drug to treat a rare muscle disease which affects some 20 children in the Netherlands ...

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PHARMAC asked to fund drug for children with spinal muscular atrophy after it changes four year old's life

5 May 2021 - Four year old Charlotte Bond couldn’t roll over by herself two years ago. ...

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NICE announces more people eligible for nusinersen sodium following review of Managed Access Agreement

4 May 2021 - NICE has today announced that more people with the rare genetic disorder spinal muscular atrophy are to ...

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TGA publishes AusPAR for Zolgensma

22 April 2021 - Rapid publication following approval. ...

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Additional analysis of Luxturna in the treatment of visual impairment caused by hereditary eye disease

21 April 2021 - TLV has made a supplementary analysis to the previous evaluation of Luxturna, as Novartis has submitted new ...

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LEXEO Therapeutics announces FDA fast track designation granted to LX1001 for the treatment of APOE4 associated Alzheimer’s disease

20 April 2021 - LX1001 is the first investigational gene therapy being evaluated to address the underlying genetics of Alzheimer’s disease. ...

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Alberta family waiting on approval for expensive, life-saving drug for young boy

23 March 2021 - Reign Johnston turned 2 on March 5. One of his grandpas and an uncle came to ...

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FDA approves first cell-based gene therapy for adult patients with multiple myeloma

27 March 2021 - The U.S. FDA approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple ...

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