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Onasemnogene abeparvovec for Spinal Muscular Atrophy: Experience from one center in the United Arab Emirates

Background Onasemnogene abeparvovec (Zolgensma) is approved by FDA, EMA and over 40 countries as the gene therapy for Spinal Muscular Atrophy. In 2020, Novartis launched Global Managed Access Program (GMAP) in countries where it didn’t receive regulatory approval.

Method We collected data of the children who received Zolgensma in Medcare Women and Children Hospital, Dubai in United Arab Emirates (UAE).

Results Two children received Zolgensma under GMAP for the first time in UAE in November 2020. By March 2022, 22 children received Zolgensma. Three were residents of UAE and others were from Iran, Ethiopia, Romania, Nepal and Turkey. 50% were from Turkey. 45% received under GMAP and rest were through crowdfunding. The youngest child was 7 months whereas the oldest one was 83 months. Children weighed between 5 kg to 14 kg. All the Turkish and Romanian children received Nusinersen before the gene therapy which was given after diagnosis to them. Two children under GMAP program needed invasive ventilation after their selection but could successfully tolerate total 8 hours off ventilation within three months and hence were eligible for the therapy. 50% were on invasive ventilation and 9% had non-invasive ventilation before infusion. Transient transaminitis and thrombocytopenia were noted in almost all the children. They responded well to adjusting the steroid dose. No thrombotic microangiopathy or bleeding was reported. Children were monitored with the help of CHOP-Intend and other scales

Conclusion: All children tolerated Zolgensma infusion well with no serious complications. CHOP intend scores showed improvement in their motor skills.
Keywords: spinal muscular atrophy, gene therapy, Zolgensma