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gene therapy

This page shows the latest gene therapy news and features for those working in and with pharma, biotech and healthcare.

Novartis publishes final positive phase 3 results of Zolgensma in Nature Medicine

Novartis publishes final positive phase 3 results of Zolgensma in Nature Medicine

Almost all children involved with copies of the SMN2 gene treated pre-symptomatically met key milestones. ... Zolgensma is currently the only approved gene therapy for the treatment of SMA and is the only SMA treatment produced to directly tackle the

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  • A creative awakening – how pharma is bringing innovation to the patient journey A creative awakening – how pharma is bringing innovation to the patient journey

    We can actually make it easier for patients to get the best from the amazing innovation, such as gene therapy and CRISPR, that is coming through.”. ... The challenge is to bring creativity into every part of the therapy and patient journey.

  • Listen, learn and lift off Listen, learn and lift off

    They were industry set-pieces that drew an audience that watched the vapour trail as a new therapy locked onto its destination. ... The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are expecting a busy year for approvals,

  • How early-stage clinical research is facilitating the new wave of personalised medicines How early-stage clinical research is facilitating the new wave of personalised medicines

    Moreover, as an industry we need to ensure that we are equipping pharmacologists with the right skills to undertake gene therapy studies. ... Lessons learned and building on successful gene therapy studies. The development of early-phase precision

  • EU cross-border healthcare EU cross-border healthcare

    The reality for many patients and their families is sobering. Gene therapies and other Advanced Therapy Medicinal Products (ATMPs) require specialist treatment processes. ... The current challenges patients face when seeking access to an approved gene

  • Rare genetic diseases – what’s in store for 2021? Rare genetic diseases – what’s in store for 2021?

    Gene therapy will continue to evolve. Gene therapy is a truly exciting field. ... However, the door has opened and many more are starting to realise the true potential that different types of gene therapy offer.

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