Search Results - "Markati, Theodora"

The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review by Poleur, Margaux, Markati, Theodora, Servais, Laurent

“…Developing drugs for rare diseases is challenging, and the precision and objectivity of outcome measures is critical to this process. In recent years, a number…”
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Lessons Learned from Discontinued Clinical Developments in Duchenne Muscular Dystrophy by Markati, Theodora, De Waele, Liesbeth, Schara-Schmidt, Urlike, Servais, Laurent

“…Duchenne muscular dystrophy (DMD) is an X-linked condition caused by a deficiency of functional dystrophin protein. Patients experience progressive muscle…”
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Therapeutic hypothermia for mild neonatal encephalopathy: a systematic review and meta-analysis by Kariholu, Ujwal, Montaldo, Paolo, Markati, Theodora, Lally, Peter J, Pryce, Russell, Teiserskas, Justinas, Liow, Natasha, Oliveira, Vânia, Soe, Aung, Shankaran, Seetha, Thayyil, Sudhin

“…To examine if therapeutic hypothermia reduces the composite outcome of death, moderate or severe disability at 18 months or more after mild neonatal…”
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Emerging therapies for Duchenne muscular dystrophy by Markati, Theodora, Oskoui, Maryam, Farrar, Michelle A, Duong, Tina, Goemans, Nathalie, Servais, Laurent

“…Duchenne muscular dystrophy is an X-linked disease caused by the absence of functional dystrophin in the muscle cells. Major advances have led to the…”
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A systematic review of adeno-associated virus gene therapies in neurology: the need for consistent safety monitoring of a promising treatment by Horton, Rebecca H, Saade, Dimah, Markati, Theodora, Harriss, Elinor, Bönnemann, Carsten G, Muntoni, Francesco, Servais, Laurent

“…Adeno-associated virus (AAV) gene therapies are generating much excitement in the rare disease field, particularly for previously untreatable neurological…”
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Early clinical and pre-clinical therapy development in Nemaline myopathy by Fisher, Gemma, Mackels, Laurane, Markati, Theodora, Sarkozy, Anna, Ochala, Julien, Jungbluth, Heinz, Ramdas, Sithara, Servais, Laurent

“…Nemaline myopathies (NM) represent a group of clinically and genetically heterogeneous congenital muscle disorders with the common denominator of nemaline rods…”
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Pre-emptive opioid sedation during therapeutic hypothermia by Montaldo, Paolo, Vakharia, Anuj, Ivain, Phoebe, Mendoza, Josephine, Oliveira, Vania, Markati, Theodora, Shankaran, Seetha, Thayyil, Sudhin

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