Alnylam announces U.S. FDA has granted priority review of the lumasiran new drug application for the treatment of primary hyperoxaluria type 1

26 May 2020 - PDUFA date set for 3 December 2020. ...

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Passage Bio receives rare paediatric disease designation for PBGM01 for patients with GM1 gangliosidosis

21 May 2020 - PBGM01 to enter the clinic in fourth quarter of 2020. ...

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B.C. teen with rare disease wins fight to receive costly drug from province

19 May 2020 - Health ministry won't explain change of heart in granting Miles Ambridge treatment for spinal muscular atrophy. ...

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AveXis receives EC approval and activates “Day One” access program for Zolgensma, the only gene therapy for spinal muscular atrophy

19 May 2020 - Zolgensma (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy ...

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Japan OKs public insurance coverage for SMA drug Zolgensma

13 May 2020 - A panel that advises the health minister approved on Wednesday coverage for Novartis' spinal muscular atrophy ...

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Japan to set price of SMA drug Zolgensma at 167 million Yen

12 May 2020 - Japan's health ministry is planning to set the price of Novartis AG's spinal muscular atrophy drug ...

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Third evaluation within the FINOSE collaboration

12 May 2020 - Together with the corresponding authorities in Finland and Norway, TLV has produced a joint evaluation report ...

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Health economic assessment of gene therapy Zynteglo in transfusion-dependent beta-thalassemia

11 May 2020 - TLV has developed a health-economic knowledge base for the Zynteglo gene therapy (autologous CD34-positive cells expressing the ...

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'New Zealand doesn't have help juice' - PHARMAC's decision leaves family stuck abroad

3 May 2020 - Kristie Yeoman says returning home would likely mean death for her daughter. ...

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Spinraza could give teen a longer life, but Government says it's too expensive

28 April 2020 - A Greymouth father is battling for PHARMAC funding for a drug he hopes can help his ...

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PHARMAC won't fund drug Spinraza to help children with spinal muscular atrophy

24 April 2020 - The parents of babies with a rare and debilitating condition will have to wait longer for ...

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AveXis receives positive CHMP opinion for Zolgensma, the only gene therapy for spinal muscular atrophy

28 March 2020 - AveXis, a Novartis company, today announced that the CHMP of the EMA has adopted a positive ...

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Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma the only gene therapy for patients with spinal muscular atrophy

19 March 2020 - Reimbursement with MHLW is expected by the end of 1H20, pending agreement, Zolgensma will be available at ...

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Gene and cell therapies continue growth in patients, financing, and costs

5 March 2020 - The number of people treated with approved gene therapies and gene-modified cell therapies like CAR-T cancer ...

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Alnylam announces approval of Givlaari (givosiran) in the European Union for the treatment of acute hepatic porphyria in adults and adolescents

3 March 2020 - Givlaari approved based on ENVISION Phase 3 study results showing a 74% reduction in the rate of ...

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