News About Angelman Syndrome
Current Clinical Trials for Angelman Syndrome
Minocycline in the Treatment of Angelman syndrome - There is mounting evidence to suggest that a treatment for Angelman syndrome is not just possible, but probable. The lack of known molecular targets associated with Angelman syndrome has hampered the development of specific therapeutics. However, a recent surge of potential therapeutics for other disorders associated with cognitive disruption has begun to be used in human clinical trials - read more
Levadopa Study - Levodopa is a prodrug that "delivers" dopamine to the brain. It is usually given with carbidopa, a peripheral decarboxyla-se inhibitor, to increase the bioavailability of levodopa. Animal studies have suggested that levodopa can reverse the excess phosphorylation of some enzymes involved in synaptic and neuronal function, including calcium/calmodulin-dependent kinase type 2 (CaMKII) - read more
Natural History Study - This study is designed to conduct longitudinal multidisciplinary investigations on the natural history, morbidity and mortality of Angelman syndrome. Detailed longitudinal data on a cohort of Angelman syndrome individuals will be collected to gain a better understanding of the disease progression, and follow the natural history of the clinical features of this patient cohort including assessment of quality of life and longevity - read more
Sleep Treatment Study - This research study will investigate sleep behavior in the rare neurological disorders Angelman syndrome, Rett syndrome and Prader-Willi Syndrome. Sleep is very important to proper health and plays a critical role in learning, memory, brain development and brain function. Sleep disturbances can have negative effects on health and the quality of life of children and their families - read more
Upcoming Angelman Syndrome Clinical Trial - Ovid Therapeutics and Lundbeck A/S pharmaceuticals have entered into a worldwide licensing agreement for Gaboxadol, a drug that Ovid plans to use in separate upcoming clinical trials to treat Angelman syndrome and Fragile X syndrome. Gaboxadol is a highly selective extrasynaptic GABA(A) receptor agonist that restores tonic inhibition at low doses and has been shown to correct the motor deficit in a mouse model of Angelman syndrome. Gaboxadol compensates for the reduced extrasynaptic GABA that results from increased levels of the GABA transporter 1 (or Gat1) observed in the Angelman syndrome mouse model. A Phase 2 clinical trial is expected to commence in 2016. - read more
ASAA Newsletter
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