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    • Genomics England

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    Havas Life London unveils AI awareness campaign

    The campaign highlights the dangers of AI misinformation in rare disease

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    FDA approves first gene therapy for Wiskott-Aldrich syndrome

    Symptoms of the rare disease include bleeding, eczema and frequent infections

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    AstraZeneca announces $2bn investment in US manufacturing

    The new and expanded facilities will support production of rare disease drug treatments

    - PMLiVE

    AstraZeneca reports positive results from rare disease trial

    HES is a group of rare disorders characterised by persistently elevated levels of white blood cells

    - PMLiVE

    NHS programme to give head and neck cancer patients fast-track access to mRNA vaccine

    More than 11,000 people are diagnosed with head and neck cancers every year in England

    Navigating NHS Market Access: Policy Change and Opportunity in England With Sarah Everest Ford

    Navigating NHS Market Access: Policy Change and Opportunity in England

    With the changes at NHS England and the publishing of the 10-Year Plan, there is a lot of talk of change in the NHS in England. But what do these...

    Petauri Evidence

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    Digital Transformation in the NHS’s 10-Year Plan: Techno-Optimism and the Limits of Efficiency

    Here, Evelyne Priestman (Senior Consultant – HEOR) and Mary Baker (Senior Medical Writer) at Petauri Evidence explore the National Health Service (NHS) in England’s newly unveiled 10-year plan through the...

    Petauri Evidence

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    Live Q&A: Beyond NHS England – Navigating changes at NHSE and DHSC

    What impact can we expect from the abolition of NHS England (NHSE) and changes at the Department of Health and Social Care (DHSC)? What do these changes mean for your...

    Petauri Evidence

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    Amgen’s Uplizna approved by FDA as first treatment for rare disease IgG4-RD

    Immunoglobulin G4-related disease affects approximately 20,000 people in the US

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    FDA approves Soleno’s Vykat XR as first treatment for hyperphagia in Prader-Willi syndrome

    The rare genetic disorder affects an estimated one in every 15,000 newborns in the US

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    Novartis’ Fabhalta granted FDA approval to treat ultra-rare kidney disease C3G

    Approximately half of C3G patients progress to kidney failure within ten years of diagnosis

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    Live Q&A: Beyond NHS England – Restructuring and its implications for Pharma and Medtech

    What does the abolition of NHS England mean for your market access and NHS Engagement strategy? How will the changes at national and integrated care board (ICB) level impact launch...

    Petauri Evidence

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