2020 Update on Gene Therapy for CNS Diseases Explore the latest advancements in gene therapy for CNS diseases with researcher Jim Wilson. Learn about challenges, clinical trials, and future directions in this comprehensive update.
Angelman Syndrome IPSC and Brain Organoid Biorepository Explore the groundbreaking project by FAST and Yale University to create a biorepository for Angelman Syndrome iPSCs and brain organoids, aiming to develop novel treatments.
Background on Neuren Pharmaceuticals and NNZ 2591 Explore Neuren Pharmaceuticals’ progress in developing drugs for neurodevelopmental disorders. Learn about their lead program, NNZ-2591, and its promising results in treating synaptic plasticity deficits.
Benefits:Challenges of Human Cerebral Organoids for Angelman Syndrome Research Explore the benefits and challenges of using human cerebral organoids in Angelman Syndrome research with Albert Keung. Discover the importance of cell diversity and genetic origins in disease understanding and therapeutic development.
Patient’s Role in Drug Development Explore David Meeker’s insights on the crucial role patients play in drug development for rare diseases, emphasizing the power of storytelling and community resilience.
Putting Patients at the Center: An Overview of Taysha Explore Taysha Gene Therapies’ mission to treat monogenic diseases of the central nervous system. Learn about their patient-centric approach, innovative gene therapy development, and commitment to collaboration.
Roche Angelman Syndrome Program Update – 2021 Explore Roche’s Angelman Syndrome Program, its progress, and the promising lead compound, UBE3A-LNA. Learn about the FREESIAS observational study and TANGELO Phase 1 drug study.
Therapeutic Strategies for the Treatment of Angelman syndrome Explore the latest therapeutic strategies for Angelman Syndrome treatment, including gene replacement therapy and paternal activation. Learn from expert Allyson Berent’s insights on clinical trials and research participation.
Updates on the Development of an ASO Therapy for Angelman Syndrome Explore the latest updates on Ionis Pharmaceuticals’ development of an ASO therapy for Angelman Syndrome. Learn about the upcoming clinical trials, patient perspectives, and how to prepare for the future.
Validation of ORCA Outcome Measure Explore Bryce Reeve’s article on the validation of the ORCA measure, a tool developed to assess communication abilities in children with Angelman Syndrome. Discover its development, testing, and implementation.
We may request cookies to be set on your device. We use cookies to let us know when you visit our websites, how you interact with us, to enrich your user experience, and to customize your relationship with our website.
Click on the different category headings to find out more. You can also change some of your preferences. Note that blocking some types of cookies may impact your experience on our websites and the services we are able to offer.
Essential Website Cookies
These cookies are strictly necessary to provide you with services available through our website and to use some of its features.
Because these cookies are strictly necessary to deliver the website, refusing them will have impact how our site functions. You always can block or delete cookies by changing your browser settings and force blocking all cookies on this website. But this will always prompt you to accept/refuse cookies when revisiting our site.
We fully respect if you want to refuse cookies but to avoid asking you again and again kindly allow us to store a cookie for that. You are free to opt out any time or opt in for other cookies to get a better experience. If you refuse cookies we will remove all set cookies in our domain.
We provide you with a list of stored cookies on your computer in our domain so you can check what we stored. Due to security reasons we are not able to show or modify cookies from other domains. You can check these in your browser security settings.
Google Analytics Cookies
These cookies collect information that is used either in aggregate form to help us understand how our website is being used or how effective our marketing campaigns are, or to help us customize our website and application for you in order to enhance your experience.
If you do not want that we track your visit to our site you can disable tracking in your browser here:
Other external services
We also use different external services like Google Webfonts, Google Maps, and external Video providers. Since these providers may collect personal data like your IP address we allow you to block them here. Please be aware that this might heavily reduce the functionality and appearance of our site. Changes will take effect once you reload the page.
Google Webfont Settings:
Google Map Settings:
Google reCaptcha Settings:
Vimeo and Youtube video embeds:
Other cookies
The following cookies are also needed - You can choose if you want to allow them:
Privacy Policy
You can read about our cookies and privacy settings in detail on our Privacy Policy Page.