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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

    Exhibition Trends 2026: What Pharma Exhibitors Need to Know Now

    Exhibitions remain one of the most powerful ways for pharmaceutical and life sciences organisations to connect, educate and build trust. Whether you're launching innovations, strengthening partnerships or showcasing research, the...

    Apex Events and Exhibitions

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    AstraZeneca announces $2bn investment in US manufacturing

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

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    AstraZeneca reports positive results from rare disease trial

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

    Biogen Idec building

    Biogen’s Qalsody granted MHRA approval to treat rare form of motor neurone disease

    Mutations in the SOD1 gene are responsible for approximately 2% of all cases of the disease

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    Next-gen Medical Education Technologies to Keep an Eye on in 2025

    Dive into 2025's cutting-edge medical education technologies like immersive training, interactive data visualization, and microlearning. Discover how to boost engagement and drive real HCP impact.

    Impetus Digital

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    Eli Lilly and Alchemab Therapeutics enter ALS agreement worth up to $415m

    The deal follows the companies’ earlier partnership to advance candidates for the neurodegenerative disease

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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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    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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