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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.

Afinitor and Uptravi gain funding from NHS England

Afinitor and Uptravi gain funding from NHS England

Rare disease treatments navigate tricky market access route. Novartis’ Afinitor in Tuberous Sclerosis Complex (TSC)-related epilepsy patients and Actelion’s pulmonary arterial hypertension (PAH) treatment Uptravi (selexipag) have gained funding

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Latest Intelligence

  • 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?’

    The initiative that shows how plasma protein therapies improve lives. Plasma proteins help people with rare diseases live full lives. ... 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

  • New faces at Lucid, 90Ten and Page & Page New faces at Lucid, 90Ten and Page & Page

    At Synergy, Bates was project director, working in rare diseases. She was also a publisher at Wounds UK and Wounds international, heading up the medical education division. ... Elaine Downey joins Vivid – Lucid’s rare and specialist diseases agency

  • 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.

  • Paris biocluster appoints Stéphane Roques as new chief Paris biocluster appoints Stéphane Roques as new chief

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

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