Nusinersen in spinal muscular atrophy: indication of considerable added benefit in children with an early onset of the disease

1 March 2021 - The treatment delays permanent ventilation of the children and extends life. In addition, the children develop more ...

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FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation

25 February 2021 - Today, the U.S. FDA granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular ...

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bluebird bio ‘baffled’ after NICE rejects beta thalassaemia gene therapy

16 February 2021 - It’s back to the drawing board for bluebird bio and its discussions with NICE, which has ...

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Alberta announces funding for pricey gene therapy drug Zolgensma

Alberta Health Minister Tyler Shandro announced funding Wednesday for the gene therapy Zolgensma, which is used to treat toddlers with ...

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MeiraGTx announces AAV-CNGA3 granted fast track designation by U.S. FDA for treatment of achromatopsia

26 January 2021 - MeiraGTx today announced that the U.S. FDA has granted fast track designation to its AAV-CNGA3 gene therapy ...

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Taysha Gene Therapies receives rare paediatric disease and orphan drug designations for TSHA-105 for the treatment of epilepsy caused by SLC13A5 deficiency

19 January 2021 - Designations reinforce unmet need for treatment options for patients with rare form of genetic epilepsy. ...

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ICER issues correction to final evidence report on new therapies for bladder cancer

15 January 2021 - After correcting an input to the cost-effectiveness model, ICER’s health-benchmark price benchmark range for nadofaragene firadenovec has ...

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Sesen Bio submits completed biologics license application to the FDA for Vicineum and has requested priority review

21 December 2020 - BLA supported by strong Phase 3 VISTA trial data and positive analytical comparability data. ...

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Newly approved treatment could save a young child in Edmonton with a rare disease. It costs 2.8 million dollars per dose.

19 December 2020 - Reign Johnston is a happy baby who enjoys painting as much as playing with toys. His green ...

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Novartis receives complete response letter from U.S. FDA for inclisiran

18 December 2020 - The U.S. FDA has not raised any concerns related to the efficacy or safety of inclisiran. The ...

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Recommendations made by the PBAC - November 2020

18 December 2020 - The recommendations from the November 2020 PBAC meeting are now available. ...

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Health Canada approves Zolgensma, the one-time gene therapy for paediatric patients with spinal muscular atrophy

16 December 2020 - Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective ...

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Though promising, gene therapies face durability and reimbursement headwinds

11 December 2020 - The promise of gene therapy is to cure diseases associated with faulty or missing genes. There’s enormous ...

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enGene receives fast track designation for EG-70 for the treatment of non-muscle invasive bladder cancer

3 December 2020 - enGene announced today that the U.S. FDA has granted fast track designation to enGene for EG-70, the ...

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Alnylam receives approval for Oxlumo (lumasiran) in the European Union for the treatment of primary hyperoxaluria type 1 in all age groups

19 November 2020 - Oxlumo is the first therapeutic approved for the treatment of primary hyperoxaluria type 1, and the ...

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