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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.

Redmile and Google help Evox raise £35.5m for exosome pipeline

Redmile and Google help Evox raise £35.5m for exosome pipeline

The cash injection – led by Redmile Group and with Google’s venture arm GV and Cowen Healthcare among the new investors – will help the company progress “several proprietary rare disease assets ... Earlier-stage programmes are looking at other

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Latest Intelligence

  • 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.

  • 30 Women Leaders in UK Healthcare

    7. Joanne Hackett, Chief Commercial Officer, Genomics England. Genomics England is that rare thing for the UK and the NHS – a clear global leader in its field, thanks to its groundbreaking ... It represents the world’s largest depository of whole

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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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Latest from PMHub

  • PRMA Consulting Ltd

    Our experience encompasses a wide range of indications, including; cardiovascular and renal, gastroenterology, immunology, infectious diseases and vaccines, metabolism and endocrinology, neurology and psychiatry, oncology/hematology, ophthalmology,

  • Comradis

    Comradis. Comradis is an independent healthcare consultancy that specializes in rare diseases and uncommon cancers.

  • Pharma Market Research Report - Predictions for 2016

    Last year the FDA approved 48 drugs in rare diseases, including both novel and repurposed drugs. ... Rare diseases are attractive to pharma for a number of reasons.

  • Vivid Medical Communications Supports the 10th Anniversary of Rare Diseases Day 2018

    Collectively, rare diseases comprise 6, 000 conditions that affect over 600 million people around the world. ... Rare Diseases Day is an encouraging time for patients with rare diseases and for Vivid, according to Alison Cantle, Vivid’ s newly

  • EphMRA 2017 round up

    EphMRA 2017 round up. We were delighted to present, exhibit and sponsor this year’ s EphMRA Annual Conference in Amsterdam, Netherlands. Director and EphMRA board member, Richard Head, Director, Jennifer Redfearn and Associate Director, An-hwa Lee

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