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Gaucher's disease

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

R&D productivity takes off

R&D productivity takes off

Among these, notable examples include PTC Therapeutics' Translarna (ataluren) - the first medicine approved for the treatment of Duchenne muscular dystrophy (DMD) - and Sanofi/Genzyme's Cerdelga (eliglustat) for Gaucher disease, a ... Moreover, pharma's

Latest news

  • Genzyme's oral Gaucher drug cleared in Europe Genzyme's oral Gaucher drug cleared in Europe

    Genzyme's oral Gaucher drug cleared in Europe. Cerdelga now available to adults with type 1 Gaucher disease. ... Patients with Gaucher disease in Europe will soon have access to a first-line, oral alternative to injectable therapy, following approval of

  • FDA gives green light to Genzyme’s oral Gaucher drug FDA gives green light to Genzyme’s oral Gaucher drug

    Sanofi's Genzyme subsidiary has won approval in the US for Cerdelga, which it says is the only first-line oral therapy for adults with type 1 Gaucher disease. ... Cerdelga, which was granted a priority review by the FDA, is “ another important

  • The moving parts of orphan drug development The moving parts of orphan drug development

    The acquisition of the US biotech brought Sanofi new treatments for multiple sclerosis, Gaucher's disease and Fabry disease. ... The company already had its own orphan brands such as Replagal (agalsidase alfa) for Fabry disease, Hunter's syndrome

  • Pharma companies back Rare Disease Day Pharma companies back Rare Disease Day

    A multitude of pharma companies, including Sanofi's rare disease division Genzyme, have leant their support to Rare Disease Day. ... In China, Genzyme will share the stories of Gaucher's disease patients with the public through photos, videos, and local

  • CHMP turns down Pfizer’s Gaucher drug Elelyso

    The European Medicines Agency's (EMA) primary advisory committee has recommended against approval of Protalix and Pfizer's Gaucher disease treatment Elelyso (taliglucerase alfa), despite concluding that the benefits of treatment ... Pfizer and Protalix

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