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Rare Diseases

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  • Trial shows chemotherapy is helping kids live with pulmonary vein stenosis

    Pulmonary vein stenosis (PVS) is a rare disease in which abnormal cells build up inside the veins responsible for carrying oxygen-rich blood from the lungs to the heart. It restricts blood flow through these vessels, eventually sealing them off entirely if left untreated. Typically affecting young c more

  • BioVaria Startup Awards go to OPSYON and LIfT Biosciences

    Over 250 innovative minds gathered in Munich for the 11th BioVaria to discuss emerging life-science trends and initiate partnerships between academia and industry. 63 peer-selected, patent-protected technologies originating from as many research institutes, universities and university hospitals from more

  • GSK transfers rare disease gene therapy portfolio

    GSK and Orchard Therapeutics announced a strategic agreement, under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard, securing the continued development of the programmes and access for patients. This acquisition strengthens Orchard’s posit more

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  • Afinitor - A Two-Pronged Attack Against Tuberous Sclerosis Complex

    Introduction GlobalData’s new alert, "Afinitor - A Two-Pronged Attack Against Tuberous Sclerosis Complex”, takes a look at the implications of the FDA’s recent approval of the drug for the treatment of angiomyolipomas, benign kidney tumors that affect a majority of people with this rare disease. more

  • United Kingdom Pharmaceuticals and Healthcare Report Q4 2012

    BMI View: We retain our broadly pessimistic outlook for the UK’s pharmaceutical market over the five years to 2016, although the emphasis on cost containment will serve to boost the performance of the generic drug sector. A difficult economic situation will also prioritise economically priced items more

  • Israel Pharmaceuticals and Healthcare Report Q2 2012

    BMI View: Following widespread protests in mid-2011, the Israeli government has been under significant pressure to spend more on housing, healthcare and education. While it was not about to tear down the country's privatised healthcare system, it has been able to support it by subsidising sick funds more

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