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Gaucher disease

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

Lilly picks up gene therapy programme in $1bn Prevail Therapeutics acquisition deal

Lilly picks up gene therapy programme in $1bn Prevail Therapeutics acquisition deal

Prevail’s pipeline spans clinical-stage and preclinical neuroscience assets, including lead gene therapies PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD) ... It has also been granted

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  • Pharma deals during November 2014 Pharma deals during November 2014

    Cerdelga, an oral therapy for adult patients with Gaucher disease type 1, was developed by Genzyme and approved by the FDA in August this year. ... BioMarin was awarded a Rare Pediatric Disease PRV when it received approval of Vimizim, a new biological

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  • Spotlight interview: 15 minutes on patient-centricity in pharma

    Another example was a project we did for Gaucher disease type 1. ... This project involved training nurses on patient activation. The nurses felt they lacked the in-depth connection with their Gaucher disease patients so were unable to make the most of

  • emotive adds to medical team with new Scientific Lead and Associate Medical Writer

    Kate is experienced in a wide range of therapy areas, including immunology, specifically Crohn’s disease, ulcerative colitis and psoriatic arthritis, in ophthalmology, including retinal diseases, glaucoma and dry eye disease, ... in rare

  • Making good practice common practice in rare disease

    2016 – Researchers found that people with Gaucher disease ascribed more importance to fatigue than to other disease parameters, while their doctors put greater weight on objective measures of visceral and hematologic ... symptoms. Literature suggests

  • 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,

  • Can rare diseases be a viable option for the pharma industry?

    An example of this would be Gaucher’s disease, which has 3 specific sub-types of patients requiring dynamic modeling of the data from patient registries over a 60 year time ... Epiomic Patient Segmentation Database, Metabolic Disorders Category,

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