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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.

Freeline raises £88m to accelerate haemophilia gene therapy

Freeline raises £88m to accelerate haemophilia gene therapy

and take another for Fabry disease into the clinic. ... This adds to Freeline and its focus on chronic systemic disease, Nightstar ( specialising in inherited retinal diseases), and Gyroscope, which specialises in retinal inflammation.

Latest news

  • Amicus Therapeutics launches Fabry treatment Galafold in Italy Amicus Therapeutics launches Fabry treatment Galafold in Italy

    US biotech Amicus Therapeutics has launched its new Fabry disease treatment Galafold (migalastat) in Italy after winning reimbursement coverage. ... Galafold is the first orally administered alternative to the current crop of injectable enzyme

  • Amicus gets NICE green light for Fabry drug Amicus gets NICE green light for Fabry drug

    The National Institute of Health and Care Excellence has given its blessing to Amicus' oral therapy for Fabry disease, a rare genetic disorder. ... Fabry disease affects an estimated 855 people in England, of whom around 140 would be eligible for

  • SMC approves Opdivo combo as first-line skin cancer treatment SMC approves Opdivo combo as first-line skin cancer treatment

    Amicus Therapeutics' Galafold (migalastat) has also received the go-ahead, and has been sanctioned by the SMC for the treatment of adults with Fabry disease, an incurable inherited genetic disorder. ... greater enzyme activity after evidence of renal,

  • NICE backs Amicus Therapeutics’ Galafold NICE backs Amicus Therapeutics’ Galafold

    Fabry disease affects an estimated 855 people in England, of whom around 140 would be eligible for treatment with Galafold. ... An incurable inherited genetic disorder, Fabry disease is caused by a faulty enzyme responsible for breaking down fatty

  • GSK gene therapy tops CHMP recommendations GSK gene therapy tops CHMP recommendations

    Using donated cells also carries a risk of graft-versus-host disease, which is bypassed using GSK's therapy. ... Amicus Therapeutics got a green light from the CHMP for Galafold (migalastat) as the first oral treatment for Fabry disease, which is caused

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Latest Intelligence

  • 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

    Firstly, GSK decided to return rights to the experimental Fabry disease drug migalastat, noting that support would continue through GSK's equity investment of $3m. ... The deal gives Amicus access to Callidus' late-stage enzyme replacement therapy for

  • 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

    Our rare disease team understands the cornerstones of rare disease education and what makes it different from other therapy areas. ... Lucid’ s Rare Disease Business Unit currently works with clients in Fabry disease, Pompe disease, Gaucher disease,

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