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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.

Daily brief: Novartis spends big on ezcema,

Daily brief: Novartis spends big on ezcema,

TMeiraGTx is its first investment to IPO. MeiraGTx has strong roots in UCL- its rare eye diseases business emerged from the labs of Professor Robin Ali, a human molecular genetics expert ... Funds raised from the IPO will enable MeiraGTx to progress its

Latest news

  • NHS England says yes to Roche's Hemlibra NHS England says yes to Roche's Hemlibra

    a rarer form of the disease), Sobi’s Kineret (anakinra) for periodic fevers and autoinflammatory diseases and Amryt Pharma’s Lojuxta (lomitapide) for homozygous familial hypercholesterolaemia (HoFH). ... He added that NHS England’s medicine review

  • Malthus’ orphans Malthus’ orphans

    By and large, rare diseases and orphan drugs don’t lend themselves to the former or the latter. ... And if prices don’t decline significantly, rare diseases, personalised therapies and other expensive niche therapies will consume much more than the

  • Sanofi’s rare blood disorder drug Cablivi set for European approval Sanofi’s rare blood disorder drug Cablivi set for European approval

    Rare disease drug among notable CHMP recommendations. Sanofi is closing in on EU approval of Cablivi for acquired thrombotic thrombocytopenic purpura (aTTP) – the lead asset behind its 9.3bn acquisition of ... The drug acts as an enzyme replacement

  • Patient engagement hygiene factors Patient engagement hygiene factors

    There are great examples of pharma rising to this challenge with engagement programmes, particularly in the field of rare diseases and HIV, that are changing old perceptions and breaking new ground.

  • Roche’s MabThera ends drug drought for serious skin disease Roche’s MabThera ends drug drought for serious skin disease

    who are currently faced with a treatment regimen that can cause significant, long-term side effects, ”according to Victoria Werth, a specialist in autoimmune skin diseases based at the University of

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Latest Intelligence

  • ‘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

  • 30 women leaders in UK healthcare (part 3) 30 women leaders in UK healthcare (part 3)

    Dr Williams also founded the GSK Non-communicable Diseases (NCD) Open Lab, an open-innovation initiative designed to stimulate and support research by African academic researchers to understand and address the ... There has been no shortage of ‘big

  • Leo has its eyes on the prize Leo has its eyes on the prize

    It has also pledged to build new facilities in translational medicine in rare skin diseases and has 25 potential biomarkers under the microscope.

  • Accelerating diagnosis in rare disease Accelerating diagnosis in rare disease

    Timely diagnosis in rare disease is one of the fundamental unmet needs for patients, healthcare professionals (HCPs) and biopharma. ... However, as referring and prescribing doctors in rare disease can be small in number and geographically spread,

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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

  • 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

  • Raising Awareness: Rare Disease Day

    hundreds of patient organizations across the world will be holding events to advocate rare diseases. ... There are approximately 5, 000– 8, 000 rare diseases.(3) The majority of these are caused by changes in genes called genetic mutations.

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Four Health Communications

Formerly known as Packer Forbes Communications, Four Health Communications is an award-winning, integrated healthcare communications agency for pharma, biotech, NGOs...

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