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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.

EMA and FDA calls for innovative approaches for Gaucher disease medicines

EMA and FDA calls for innovative approaches for Gaucher disease medicines

EMA and FDA calls for innovative approaches for Gaucher disease medicines. ... disorder Gaucher disease.

Latest news

  • FDA changes its mind on 23andMe's genetic testing kits FDA changes its mind on 23andMe's genetic testing kits

    Along with Alzheimer's and Parkinson's, the new Genetic Health Risk (GHR) test covers coeliac disease, alpha-1 antitrypsin deficiency, early onset primary dystonia, Factor XI deficiency, type 1 Gaucher ... tests. The exemption will not however include

  • Langland and CAHG take top prizes at Best of Health Awards Langland and CAHG take top prizes at Best of Health Awards

    Healthcare Professional - Direct Marketing &Promotional Communications Individual. 'Hard to see, but easy to find - Rare Disease Awareness Campaign for Gaucher Disease' for Shire Pharmaceuticals, by bmore Creative .

  • Shire picks winner for Gaucher film contest Shire picks winner for Gaucher film contest

    A Shire spokesperson told PMLiVE: “ We are hoping the videos are seen by the Gaucher community, and also by the general public, to raise awareness of Gaucher disease.”. ... At Shire, we are committed to working with the community to raise awareness

  • Patient survey finds pharma falls short on drug pricing Patient survey finds pharma falls short on drug pricing

    PatientView highlights that the company ranked number one for corporate reputation - Sanofi - has a strong track record in developing innovative medicines for diseases with unmet needs, such as Gaucher disease and

  • Shire rumoured to be pursuing Actelion Shire rumoured to be pursuing Actelion

    Shire is considering a move to acquire Swiss biopharma company Actelion in a move that would boost its rare disease portfolio. ... Buying Actelion would give Shire a portfolio of drugs to treat pulmonary artery hypertension (PAH) including Tracleer

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Latest Intelligence

  • 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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Latest from PMHub

  • Key Factors Contributing Towards Growth of the Mucopolysaccharidosis Treatment Market During 2017-2027

    The Orphan Disease Center is offering MPS I Pilot Grant Programs for improving the therapies for people suffering from this syndrome. ... In 2016, State of Oregon expanded the class of covered therapies to include drugs for Gaucher's disease, MPS I, MPS

  • 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

    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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