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    Home » Abu Dhabi uses breakthrough gene therapy treatment for spinal muscular atrophy
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    Abu Dhabi uses breakthrough gene therapy treatment for spinal muscular atrophy

    GetMediaExposure.com Editorial TeamBy GetMediaExposure.com Editorial TeamDecember 30, 2025No Comments3 Mins Read
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    Abu Dhabi Leads ITVISMA Therapy
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    Abu Dhabi turned into one of the first cities worldwide to utilize ITVISMA ( onasemnogene abeparvovec , the advancement genetics therapy treatment created by Novartis for back muscle atrophy (SMA).

    ITVISMA is a single gene therapy specifically developed to target the hidden hereditary source of SMA in individuals aged 2 years and older with a confirmed SMN 1 genetics mutation. ITVISMA changes the missing SMN 1 genetics to boost electric motor feature, lowering the requirement for continuous treatments needed by other therapies.

    Sheikh Khalifa Medical City (SKMC), part of SEHA and a subsidiary of PureHealth, effectively administered the therapy under the supervision of the Division of Health And Wellness– Abu Dhabi (DoH).

    World’s very first ITVISMA SMA treatment

    Abu Dhabi therefore became the first city on the planet to provide the treatment, reinforcing its condition as a leading international healthcare destination and demonstrating its commitment to person treatment through genomics and accuracy medication.

    The cutting-edge treatment obtained accelerated approval in the UAE on 25 November this year, a day after it was authorized by the US Food & & Medication Administration (FDA). This placed the UAE amongst the initial nations internationally, adhering to the USA, to back this pioneering therapy.

    At the time of the FDA approval, Novartis stated: “The authorization of ITVISMA is based on data from the registrational Stage lll STEER research and sustained by the open-label Phase lllb stamina research.

    “Itvisma revealed statistically considerable renovations in electric motor function and stabilisation of electric motor capabilities generally not seen in the natural history of the disease, with results suffered over 52 weeks of follow-up.

    “Additionally, ITVISMA demonstrated a safety and security account with adverse occasions that were consistent across both research studies. One of the most usual negative occasions in the STEER research study were upper breathing system infection and pyrexia, and the most usual negative events in the STRENGTH research were common cold, pyrexia, and vomiting.”

    Dr Noura Khamis Al Ghaithi, Under-Secretary of DoH, claimed: “This turning point shows Abu Dhabi’s commitment to delivering world-class treatment and reinforcing its placement as an international leader in health care driven by genomics and accuracy medicine.

    “By administering ITVISMA, we are proud to be amongst the initial to give this cutting-edge treatment, additional strengthening our role as a leader and accelerator in sophisticated and cutting-edge healthcare.”

    Bader Al Qubaisi, Ceo at SKMC, included: “Supplying the globe’s very first ITVISMA therapy at SKMC is a testimony to Abu Dhabi’s incorporated medical care environment under the management of the Division of Health And Wellness– Abu Dhabi. In accordance with PureHealth’s vision and DoH’s advice in defining seamless treatment pathways, and facilitating collaboration with partners such as Novartis, has enabled us to bring this life-changing treatment to our people safely and efficiently.”

    Mohamed Ezz Eldin, Head of GCC Collection at Novartis, stated: “By functioning carefully with the Department of Wellness– Abu Dhabi, and SKMC, we are proud to support accelerated accessibility to breakthrough treatments such as ITVISMA, and to add to Abu Dhabi’s expanding function as a regional and global reference for sophisticated neuromuscular treatment.”

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