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Research News & Updates

2019 - 2020 Updates

Summary of Global Research


  • Ionis Pharmaceuticals - their ASO will be headed to clinical trial in the second half of 2020

  • Roche Pharmaceuticals/Genentech - their gene modifying therapy will be headed to clinical trial in 2020

  • Researchers from UC Davis - there was full behavioral rescue in both newborn and adult mice with Angelman syndrome. This was using a human gene therapy candidate that should be ready for clinical trials in the next 12 months.⁠

  • Neuren Pharmaceuticals, Inc. - their drug NNZ-2591 will begin phase 1 trials in Australia with Phase 2 trials in the US in the second half of 2020

  • GeneTx, in partnership with Ultragenyx, Inc,. - their gene altering ASO therapy will be headed to clinical trial in the first half of 2020.

  • Ovid Therapeutics - clinical trials to evaluate the pharmacokinetics (PK), safety and tolerability of OV101 in adolescents diagnosed with Angelman syndrome or Fragile X syndrome. OV101 (gaboxadol) is a delta (d)-selective GABAA receptor agonist. The phase 3 Neptune trial was unsuccessful. Update here.


   With so many potential clinical trials for 2020, make sure you have signed up on the Global Angelman Registry!




AS Clinical Research-focused Websites

Global News Articles

Research projects - NZ and Australia

2019 - Neuren Pharmaceuticals is based in Melbourne, Australia. Lead researcher and associate professor at University of Auckland Centre for Brain Research, Dr. Jian Guan, has been involved with this research. The U.S. Food and Drug Administration (FDA) granted orphan drug status to candidate therapy NNZ-2591 to treat Angelman syndrome and two other childhood neurodevelopmental disorders for which there are no currently approved medications. NNZ-2591 went into clinical trials in 2020. See press release.

2018 - Laurie McLay, Senior Lecturer at the University of Canterbury and Associate Professor Karyn France are carrying out a research project that investigates the effectiveness of treatments for sleep disturbance in children with Rare Genetic Neurodevelopmental Disorders (RGND). The Angelman Network is supporting this study with a small grant and helping to source families to participate in the study. 


2017 - Hemant Thakker has done his Doctoral Research on ‘When I am no longer alive – understanding the wishes, worries and support needs of parents of severely disabled adults.’ The findings of this study will assist in making useful recommendations to policy makers in NZ on designing service models that are both efficient (in terms of cost) and effective in terms of their usefulness to meet the needs of severely disabled adults and their families. The Angelman Network supported this study with a small grant and helped source families to participate.  Here is the link to his final paper on Succession Planning. 


Angelman Literacy Project (ALP) 

2015 - The Angelman Network was one of the official sponsors for the Angelman Literacy Project’s 2015 summer road trip – a series of workshops run by Erin Sheldon and held across the USA.  The aim was to provide important source material and research data on Angelman syndrome and Communication. The Angelman Network assisted with the informative publication, Angelman Syndrome for Educators, authored by Erin Sheldon.

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