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Emirati twins with rare disorder get life-saving gene treatment

Emirati twins with rare disorder get life-saving gene treatment
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Abu dhabiHussein matlikMona aliRashid al hasaniAmro el saddikBurjeel hospitalமோனா அலிரஷித் அல் ஹசானிஅம்ரோ எல் சதிக்பர்ஜீல் மருத்துவமனை

Abu Dhabi s Burjeel Hospital provides transformational gene therapy

Abu Dhabi’s Burjeel Hospital provides transformational gene therapy ABU DHABI, 26 days ago VPS Healthcare’s Burjeel Hospital has successfully administered advanced gene replacement therapy, in an effort to save the lives of seven-month-old Emirati twins. With the gracious support of the government and leaders of the UAE, the therapy has been carried out on the twins by the Abu Dhabi based hospital who diagnosed them with a rare and most severe form of Spinal Muscular Atrophy – Type 1 (SMA1), a highly fatal disease. After visiting Burjeel Hospital for a neurological assessment with Consultant Pediatrician and Child Neurologist, Dr Hussein Matlik, the twin babies were diagnosed with the rare and devastating monogenetic neuromuscular disease, SMA1, which is also known as Werdnig Hoffman disease. SMA is caused by a defective or missing survival motor neuron (SMN1) gene. The genetic disorder affects nerve cells that control voluntary muscles of the body resultin

Mona aliKarim smairaWerdnig hoffmanAbu dhabiHussein matlikRashid al hasaniAbu dhabi burjeelAmro el saddikTradearabia news serviceBurjeel hospitalSpinal muscular atrophyConsultant pediatricianChild neurologistExcellence directorPutting patientsMiddle east trade news

Twins suffering from rare disease get treatment

Twins suffering from rare disease get treatment 01 Mar 2021 Mariecar Jara-Puyod, Senior Reporter An Emirati couple in Abu Dhabi is grateful for the support they have received, giving their seven-month old identical twins, found to be suffering from a rare disease, the chance to live and enjoy life. Rashid Al Hasani and Mona Ali’s sons underwent a one-and-a half hour (10:30 a.m. to 12 noon) infusion of the advanced gene therapy replacement known generically as Onasemnogene Abeparvovec (AVXS-101) on Feb. 24 (Wednesday) at Burjeel Hospital in Abu Dhabi. They had earlier sought consultations with consultant paediatrician/child neurologist Dr. Hussein Matlik who had recommended a genetics test. The test diagnosis was Werdnig-Hoffman Disease.

Barry russmanMona aliJohann hoffmanAbu dhabiHussein matlikMariecar jara puyodGuido werdnigOnasemnogene abeparvovecRashid al hasaniOregon health sciences universityNational organisation for rare diseasesBurjeel hospitalWerdnig hoffman diseaseNational organisationRare diseasesShriners hospital

Emirati twins given fighting chance after gene therapy treatment

Emirati twins given fighting chance after gene therapy treatment
thenationalnews.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from thenationalnews.com Daily Mail and Mail on Sunday newspapers.

Hussein matlikMona aliRashid al hasaniAmro el saddikSeven month old emiratiBurjeel hospitalமோனா அலிரஷித் அல் ஹசானிஅம்ரோ எல் சதிக்பர்ஜீல் மருத்துவமனை

Emirati twin infants get gene replacement therapy in Abu Dhabi to correct genetic condition

Abu Dhabi: A pair of seven-month-old Emirati twins have successfully received advanced gene replacement therapy at an Abu Dhabi hospital to treat their highly fatal genetic condition. The twins were born with Spinal Muscular Atrophy Type 1 (SMA1), a rare and devastating monogenetic neuromuscular disease, also known as Werdnig Hoffman disease, which is caused by a defective or missing survival motor neuron gene (SMN1). The disorder affects the voluntary muscles of the body, resulting in rapid and irreversible loss of motor neurons, which then affects all muscle functions, including breathing, swallowing and basic movement. Over time, the condition leads to progressive muscle weakness, paralysis and even death if left untreated.

Abu dhabiHussein matlikMona aliWerdnig hoffmanRashid al hasaniAmro el saddikImage creditSpinal muscular atrophyBurjeel hospitalமோனா அலிரஷித் அல் ஹசானிஅம்ரோ எல் சதிக்படம் கடன்முதுகெலும்பு தசை அட்ரோஃபீபர்ஜீல் மருத்துவமனை