Patisiran sodium in hereditary transthyretin amyloidosis: less benefit than vutrisiran

1 March 2024 - In 2023, IQWiG found an additional benefit of vutrisiran compared to patisiran sodium based on a study.  ...

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Vutrisiran in hereditary transthyretin amyloidosis: hint of minor added benefit

17 January 2023 - Vutrisiran is approved for the treatment of patients with hereditary transthyretin amyloidosis with stage 1 or ...

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Bluebird Bio has serious cash flow concerns; inability to strike reimbursement deals in Europe partly to blame

3 April 2022 - Bluebird bio is a highly innovative biotechnology company which develops gene therapies for several rare genetic ...

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Register study on gene therapy with Zolgensma starts

1 February 2022 - Experience from clinical practice with Zolgensma, a gene therapy against certain forms of spinal muscular atrophy in ...

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EU regulation passed: G-BA works on European HTA assessment

14 December 2021 -  Today the EU Parliament passed the EU-HTA regulation for a joint health technology assessment, which includes, ...

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Drugs against rare diseases: Libmeldy gene therapy shows additional benefits - Zolgensma cell therapy, on the other hand, does not

4 November 2021 - The Federal Joint Committee (G-BA) today assessed the additional benefit of two active ingredients against very rare, ...

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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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Germany first in EU to get Novartis’ SMA gene therapy, costing almost 2 million euros

24 June 2020 - Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal ...

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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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Europe responding to gene therapy challenge, but picture remains fragmented

20 May 2019 - Germany, Italy and England using outcomes-based deals. ...

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