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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

    EMA reaffirms guidance on paracetamol use during pregnancy

    The guidance follows recent US comments about a possible link to autism

    - PMLiVE

    Johnson & Johnson seeks first EMA approval for icotrokinra for plaque psoriasis

    Icotrokinra is a first-in-class, once-daily tablet for moderate-to-severe plaque psoriasis

    - PMLiVE

    EMA orphan drug designation given to ReAlta for graft-versus-host disease therapy

    This follows preliminary results from the company's ongoing phase 2 trial

    - 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

    Organon/Henlius announce EMA validation for Perjeta biosimilar HLX11

    The reference medicine holds approvals for multiple HER2-positive breast cancer indications

    - 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

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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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