ABU
DHABI, UAE, Aug. 2, 2024
/PRNewswire/ -- The Department of Health – Abu Dhabi (DoH), the regulator of the
healthcare sector, has provided revolutionary gene therapy for
Duchenne Muscular Dystrophy (DMD) for the first time in
Abu Dhabi and outside the United States.
While previously the therapy was accessible only in the United States, this groundbreaking
treatment will now also be offered at Sheikh Khalifa Medical
City (SKMC), part of SEHA; a subsidiary of PureHealth, further
cementing Abu Dhabi's position as
a leading healthcare destination and life sciences hub globally.
The treatment was approved by the U.S. Food and Drug Administration
(FDA) in June 2023, and it was
administered to an Emirati DMD patient by a specialist team led by
a consultant paediatric neurologist from SKMC, in collaboration
with the Department's Research and Innovation Centre on March
19th 2024.
DMD is a hereditary neuromuscular disorder that causes the
muscles to gradually weaken over time. Through this
treatment, DoH and the SKMC medical team used Delandistrogene
moxeparvovec; a one-time injection that has proven to be effective
in addressing the underlying cause of DMD. The treatment can
potentially transform the disease trajectory by delivering
functional dystrophin genes into patient cells which help the body
produce biologically active dystrophin protein for muscle function,
improving muscle strength and the patient's quality of life.
Following the FDA's announcement of expanding the Delandistrogene
moxeparvovec treatment to children four and above, DoH remains
committed to providing comprehensive care and support to affected
patients and their families.
H.E. Dr. Noura Khamis Al
Ghaithi, Undersecretary of Department of Health –
Abu Dhabi (DoH),
said: "This medical milestone underscores Abu Dhabi's continuous dedication to adopting
world-class healthcare innovations that create a distinguished and
holistic healthcare experience to all members of the community and
beyond. We are committed to continuing to bring advanced
preventative, diagnostics and therapeutic capabilities to the
region, reinforcing Abu Dhabi's
position as a leading healthcare and medical tourism
destination".
Dr. Omar Ismayl, Head of
Paediatric Neurology and Consultant at SKMC, said: "We at the
Paediatric Neurology Department at SKMC are thrilled to use the
gene therapy delandistrogene moxeparvovec for the first-time in
Abu Dhabi. At SKMC, we are always
committed to providing our patients with the most advanced,
evidence-based management and treatment. This gene therapy
underscores SEHA's commitment to providing access to the world's
most advanced treatments and technologies, aligning with
PureHealth's mission to unlock the science of longevity. It
has the potential to change the unpleasant natural history and
progression of this condition. This achievement would not have
happened without the immense hard work and support from all the
staff at SKMC and DoH."
DMD results from variations in a muscle protein called
dystrophin, which normally maintains the integrity of muscle cells.
When variations in this protein arise, muscle fibres break down,
leading to a gradual weakening of the muscles that is eventually
fatal due to cardiac or respiratory failure. Research indicates
that DMD almost always affects males and is very rare in females.
With an average life expectancy of 30 years, it affects an
estimated one in 3,500-5,000 boys worldwide.
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SOURCE The Department of Health - Abu
Dhabi