Novartis CEO: gene therapy offers hope of cures in one treatment, but US needs new pricing and payment model

17 May 2019 - We live in an incredible era of progress in human health. Advances in cell and gene ...

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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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Instalment payments could be used across gene therapy space, Spark CEO says

16 May 2019 - In a fireside chat at the recently concluded Veeva Summit in Philadelphia, Jeff Marrazzo said payment over ...

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NHS chief hails SMA drug deal – and was that a swipe at Vertex?

15 May 2019 - Children in England with the rare muscle wasting disease spinal muscular atrophy will get access to ...

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NICE recommends first ever treatment for children with rare muscle wasting condition

15 May 2019 - Children with the rare genetic disorder spinal muscular atrophy can now be treated with nusinersen after NICE ...

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Homology Medicines announces FDA fast track designation for HMI-102 gene therapy development candidate for adults with PKU

14 May 2019 - Company provides details of pheNIX, the first gene therapy trial for PKU, and remains on track to ...

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Novartis pitches discounts on pricey gene therapy for deadly muscle disorder

11 May 2019 - Novartis is offering price discounts in negotiations with U.S. health insurers on its gene therapy for spinal ...

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Novartis confident of Zolgensma supply, calls $2 million price 'speculation'

8 May 2019 - Novartis is confident it has adequate production capacity for its Zolgensma gene therapy should regulators this ...

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A $2 million drug is about to hit the market

8 May 2019 -Insurers, drug makers grapple with new payment models for gene therapies that can cure diseases in one ...

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Akcea and Ionis announce approval of Waylivra (volanesorsen) in the European Union

7 May 2019 - Akcea Therapeutics and Ionis Pharmaceuticals announced today that Waylivra has received conditional marketing authorisation from the ...

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SMA patients in Saskatchewan, Canada, gain wider access to Spinraza

1 May 2019 - The Canadian province of Saskatchewan has joined that of Quebec in granting access to Spinraza (nusinersen) ...

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FDA's efforts to advance the development of gene therapy

1 May 2019 - Gene therapy has been on the horizon for several decades and has now become a reality in ...

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SMA petition: family forced to move to Australia to get daughters treated

2 May 2019 - A family have moved to Australia in order to get their daughters the treatment they need. ...

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Akcea steals march on Alnylam with NICE approval

16 April 2019 - Advantage to Tegsedi over Onpattro. ...

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Kiwi father's emotional plea to Jacinda Ardern to fund life saving drug for rare disease

15 April 2019 - The parents of two young girls diagnosed with a rare disease have urged Prime Minister Jacinda ...

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