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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

    Cerdelga (eliglustat) has been cleared by the European Commission (EC) for adults living with Gaucher disease type 1, according to Genzyme's parent Sanofi, which says the drug "will be available ... Cerdelga was first approved in the US last August when

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

    Cerdelga, which was granted a priority review by the FDA, is “another important treatment option” for Gaucher disease, according to the agency's deputy director of the Office for Drug Evaluation ... Patients with Gaucher disease accumulate lipid

  • 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

    Genzyme, Shire and more hold global campaigns today. 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

  • Orphans under the spotlight

    patients. Where medicines are used as part of a service commissioned by the NCG (for example, Genzyme's Cerezyme in the treatment of the inherited metabolic disease Gaucher's), the medicine ... Additionally, in June 2009, the UK, in common with other EU

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