The UAE-based Medcare Women and Children Hospital has successfully administered a pioneering intrathecal gene therapy for ...

A campaign group for people with a rare genetic muscular condition has called for all newborn babies to be tested for the disease. Spinal Muscular Atrophy (SMA) affects around six babies in Ireland ...

Abu Dhabi’s Sheikh Khalifa Medical City has become the first hospital globally to administer ITVISMA, a one-time gene therapy ...

The Health Information and Quality Authority (Hiqa) has begun assessing the addition of spinal muscular atrophy (SMA) to the newborn health-screening process. SMA is a rare genetic condition that ...

Living with a rare disease can be incredibly isolating, not just for those affected but for their families as well. For individuals with Spinal Muscular Atrophy (SMA), a genetic condition that weakens ...

Findings from two clinical trials suggest Novartis' Itvisma is safe and effective for SMA patients over 2 years old.

Novartis’ ITVISMA, approved a month ago by the US FDA, used at Sheikh Khalifa Medical City under the supervision of the ...

Regulator approves Itvisma for adults and children aged two and above, positioning the UAE among the first countries globally to authorise the treatment for a wider age group ...

SMA is inherited in an autosomal recessive manner, meaning a child usually inherits two mutated genes (SMN1 gene), one from each parent, to develop the disorder. Parents who carry one mutated gene are ...

Inspired by this case, Health Minister Dinesh Gundu Rao has announced plans to advocate for including SMA under the financial aid scheme for rare, high-cost diseases affecting Scheduled Caste and ...

Abu Dhabi marks a landmark achievement by becoming the first in the world to deliver ITVISMA (onasemnogene abeparvovec) for ...

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