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    AstraZeneca/Ionis’ Wainzua granted EC approval to treat rare disease ATTRv-PN

    ATTRv with polyneuropathy affects up to 40,000 people worldwide

    - PMLiVE

    AstraZeneca receives MHRA approval for Wainzua in rare nerve disease ATTRv-PN

    The therapy is designed to reduce the effects of the disease by lowering the production of TTR protein

    - PMLiVE

    Arrowhead’s RNAi therapeutic plozasiran shows promise in familial chylomicronaemia syndrome

    There are currently no therapies in the US specifically approved to treat the rare genetic disorder

    - PMLiVE

    Ionis’ investigational antisense therapy shows promise in Angelman syndrome

    ION582 is expected to enter late-stage development for the rare neurodevelopmental disorder in 2025

    - PMLiVE

    MHRA approves TMC Pharma’s Nulibry to treat rare genetic disease MoCD type A

    Molybdenum cofactor deficiency is estimated to affect one in 100,000 to 200,000 newborns worldwide

    - PMLiVE

    Ionis shares positive late-stage results for olezarsen in familial chylomicronaemia syndrome

    The rare genetic disease is estimated to affect up to 13 people per million in the US

    - PMLiVE

    FDA approves Orchard’s Lenmeldy gene therapy for metachromatic leukodystrophy

    The rare genetic disease is estimated to affect one in every 40,000 people in the US

    - PMLiVE

    Sanofi presents positive results for Pompe disease enzyme replacement therapy

    The rare genetic disorder is estimated to affect around one in every 40,000 people in the UK

    - PMLiVE

    Regenxbio’s investigational gene therapy shows promise in Hunter syndrome

    The rare genetic disease is estimated to affect one in every 100,000 to 170,000 births

    - PMLiVE

    Ionis shares positive phase 3 results for hereditary angioedema drug candidate

    More than 20,000 people in the US and Europe are affected by the rare genetic disease

    - PMLiVE

    Genomics England receives government funding for cutting-edge genomics research

    £105m will be used to accelerate the diagnosis of rare genetic diseases in newborns

    - PMLiVE

    Ionis reports positive phase 2b results for fesomersen in thrombosis prevention

    The company also announced it will regain rights to the antisense medicine from Bayer

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