Abu Dhabi announces first gene transfer therapy for duchenne muscular dystrophy

Department of Health – Abu Dhabi (DoH) has provided revolutionary gene transfer therapy for Duchenne muscular dystrophy (DMD) for the first time in the emirate. The treatment was carried out at Sheikh Khalifa Medical City (SKMC), part of SEHA, a subsidiary of PureHealth, reflecting Abu Dhabi’s continual growth as a leading destination for healthcare and life sciences globally.

Sheikh Khalifa Medical City successfully administered the treatment, in partnership with Roche Pharma, to emirates' first DMD patient, following the treatment which was only available in the US. The UAE National patient received the medication under the supervision of a specialised medical team led by a consultant paediatric neurologist.

DMD is a debilitating genetic neuromuscular disorder characterised by the gradual degeneration and weakening of muscles. Delandistrogene moxeparvovec is a one-time injection that has been proven to address the underlying cause of DMD. 

The Delandistrogene moxeparvovec treatment is provided to children between the ages of four and five who have a confirmed mutation in the DMD gene, however, it cannot be administered to children who have any deletions in exon eight and/or exon nine of the DMD gene.

Dr Asma Ibrahim Al Mannaei, Executive Director of the Research and Innovation Centre at at DoH said: “The availability of Delandistrogene moxeparvovec in Abu Dhabi has the potential to greatly enhance DMD patient outcomes and creates valuable research opportunities to further explore therapeutic interventions, enriching medical expertise regionally and internationally.”