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rare diseases

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

Orphan drugs from Biogen and Amicus win UK Prix Galien

Orphan drugs from Biogen and Amicus win UK Prix Galien

Galafold (migalastat) is an oral medication for the treatment of Fabry disease, a rare lysosomal metabolic storage condition. ... The success of products for rare disease – which, for the first time, dominated the shortlist of finalists – shows that

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  • Stakeholder dialogue in Europe’s adaptive pathways pilot Stakeholder dialogue in Europe’s adaptive pathways pilot

    Single-arm studies in rare diseases for comparison to outcomes inferred from disease registries.

  • The European Medicines Agency: PRIME’d for access? The European Medicines Agency: PRIME’d for access?

    30 PRIME treatments are in rare diseases, and16 for paediatric patients. · ... It’s hoped it could be a big step in treatment for the genetically-inherited and rare wasting disease.

  • Trust me, I’m from pharma Trust me, I’m from pharma

    They recognise these groups are becoming very powerful – particularly in rare diseases – and that their opinion matters. ... Above brand’programmes remain rare, but where they exist, they’re proving highly beneficial.

  • Bluebird Bio: on the cusp of a gene therapy revolution Bluebird Bio: on the cusp of a gene therapy revolution

    The US biotech company aims to have four groundbreaking gene therapies launched by 2022 – with the promise of lives transformed in each of these rare blood diseases – and realistic hope of ... pancreatitis. Then GSK’s Strimvelis followed in 2016

  • ‘How is your day?’ ‘How is your day?’

    Plasma proteins help people with rare diseases live full lives. Why is the value and unique aspect of these therapies not better known? ... Physicians need to be more aware of rare diseases that may hide behind recurring issues that some patients have.

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Latest appointments

  • Weekly industry appointments Weekly industry appointments

    Appleby, who will succeed CGT Catapult’s first CSO Johan Hyllner, currently serves as chief scientific officer for cell and gene therapy at GlaxoSmithKline’s (GSK) rare diseases unit.

  • Medicen Paris Region appoints new CEO Medicen Paris Region appoints new CEO

    He has also chaired the start-up Genosafe and launched the Institute of Biotherapies for Rare Diseases.

  • Gamida Cell strengthens leadership team Gamida Cell strengthens leadership team

    Their combined experience in corporate strategy, business development and commercialisation will be invaluable to the company’s continued growth as we advance our clinical programmes in blood cancers and rare genetic ... diseases.”.

  • Sobi poaches Pfizer’s Fredrick Wetterlundh Sobi poaches Pfizer’s Fredrick Wetterlundh

    supporting our organisation and our journey to become a global leader in rare diseases.”.

  • emotive bolsters client services team emotive bolsters client services team

    During her six years at Chameleon, she was responsible for medical communications acitivites in various therapy areas including diabetes, cardiology, immunology, gastroenterology and rare diseases.

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Conversis is a translation company specialising in translation and localisation for the Life Science and Pharmaceutical industry, with particular focus...

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