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Fabry disease

This page shows the latest Fabry disease news and features for those working in and with pharma, biotech and healthcare.

Sangamo surges on data for haemophilia A gene therapy

Sangamo surges on data for haemophilia A gene therapy

All three companies are chasing one-shot therapies for haemophilia A that they hope will free patients with the disease from having to take regular injections of recombinant factor VIII replacement ... reduces risk in the company’s other gene therapy

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  • Pharma deals in August 2015 Pharma deals in August 2015

    380 which will be used to treat cachexia, a wasting disease seen in cancer patients. ... Acquisition of remaining rights. 1034. Scioderm. Amicus. Galafold [migalastat] for Fabry's disease.

  • Pharma deals during November 2013 Pharma deals during November 2013

    A perfect strategic fit, this addition will create a $2bn rare disease revenue base in 2014, approx. ... Firstly, GSK decided to return rights to the experimental Fabry disease drug migalastat, noting that support would continue through GSK's equity

  • Payer focus in the personalised world of oncology and orphan diseases Payer focus in the personalised world of oncology and orphan diseases

    Personalised medicine is transforming disease understanding and many traditional diagnoses may be fundamentally revised based on the new scientific understanding. ... The Netherlands recently tried to withdraw public funding for late onset Fabry Disease

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Latest appointments

  • Sobi names Holly May VP of sales and marketing, US Sobi names Holly May VP of sales and marketing, US

    Rare disease firm Sobi has appointed Holly May as its new vice president of sales and marketing for the US. ... May has had more than 20 years’ experience in biopharmaceutical sales and marketing, with her most recent role serving as head of US

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Latest from PMHub

  • Focus on Rare Diseases

    As pharma becomes more focused on rare disease therapies, Lucid Group recognises that medical education agencies need to respond. ... Lucid’s Rare Disease Business Unit currently works with clients in Fabry disease, Pompe disease, Gaucher disease,

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