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    • CHAPLE disease

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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

    - 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

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    Regeneron’s Lynozyfic granted FDA accelerated approval to treat multiple myeloma

    More than 36,000 new cases of the blood cancer are expected to be diagnosed in the US this year

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    FDA unveils new voucher programme to accelerate drug review process

    The programme is designed to reduce the review time for companies supporting US national interests

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    Regeneron gains rights to Hansoh’s GLP-1/GIP receptor agonist in deal worth $2bn

    The candidate is being evaluated in a phase 3 obesity trial and phase 2b diabetes study

    regeneron headquarters

    Regeneron to acquire human genetics company 23andMe for $256m

    The agreement comes two months after 23andMe filed for bankruptcy protection

    - 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

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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

    - 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

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