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
Click here to download our latest newsletter - March 2018. Previous Newsletters

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Contact your State Representative here

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Our Mission

The ASAA was established to support, inform, educate, network, promote research and to advocate for families affected by Angelman syndrome - read more


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PROUDLY COLLABORATING WITH FAST AUSTRALIA 

FAST Australia
The Foundation for Angelman Syndrome Therapeutics Australia (FAST Australia) is an organisation of families and professionals dedicated to funding research to provide treatments that will improve the symptoms of Angelman syndrome and ultimately that will provide a cure (see website).