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    • Alpha-Mannosidosis

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    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

    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

    GSK’s Blenrep combination recommended by NICE to treat multiple myeloma

    The decision means that eligible patients in England will be the first in the world to access the combination

    - PMLiVE

    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

    - PMLiVE

    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

    - PMLiVE

    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

    - PMLiVE

    Novartis shares positive phase 3 results for spinal muscular atrophy gene therapy

    The company said it is planning to file regulatory applications for OAV101 IT this year

    - PMLiVE

    NICE recommends Pharming’s Joenja as first treatment for ultra-rare immune disease APDS

    Approximately 40 to 50 people in the UK are known to be living with the inherited disorder

    - PMLiVE

    Amgen’s Uplizna shows promise in phase 3 generalised myasthenia gravis study

    Regulatory submissions are expected to be complete in the first half of this year

    - PMLiVE

    NICE recommends Gideon Richter’s Ryeqo as first daily pill for endometriosis

    Around 1,000 patients could benefit from the relugolix combination therapy every year

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