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    • Castleman’s disease

    - PMLiVE

    Havas Life London unveils AI awareness campaign

    The campaign highlights the dangers of AI misinformation in rare disease

    - PMLiVE

    FDA approves first gene therapy for Wiskott-Aldrich syndrome

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

    - PMLiVE

    FDA launches TEMPO digital health pilot

    Digital technologies can improve remote patient care for those with chronic diseases

    - PMLiVE

    Richard Pazdur, FDA’s drug evaluation chief, to leave FDA just weeks into new role

    Pazdur was previously head of the FDA’s Oncology Center of Excellence

    - PMLiVE

    Merck KGaA given FDA Fast Track designation for generalised myasthenia gravis drug

    Cladribine capsules could become the first ever oral treatment for gMG

    - PMLiVE

    AstraZeneca announces $2bn investment in US manufacturing

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

    - PMLiVE

    J&J receives positive NICE recommendation for multiple myeloma treatment

    The treatment is approved for use within the NHS in England and Wales

    - 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

    Johnson & Jonson secures FDA approval for multiple myeloma treatment

    Support for first and only high-risk smouldering multiple myeloma treatment came from positive phase 3 trial results

    - PMLiVE

    GSK’s Blenrep approved by FDA for multiple myeloma

    Approval supported by positive results from phase 3 trial

    - PMLiVE

    MHRA and FDA expand collaboration on AI and medtech regulation

    National Commission on the Regulation of AI in Healthcare brings together UK and US experts

    - PMLiVE

    Roche’s small cell lung cancer therapy approved by FDA

    This is the first and only approved combination therapy for extensive-stage small cell lung cancer

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