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What is Angelman syndrome? (AS)
Angelman syndrome (AS) is a rare genetic condition that affects the nervous system, leading to challenges with movement, balance, and communication. It is caused by changes to chromosome 15, which impact brain function.
What is Angelman syndrome? (AS)
Angelman syndrome (AS) is a rare genetic condition that affects the nervous system, leading to challenges with movement, balance, and communication. It is caused by changes to chromosome 15, which impact brain function.
We start with the barrier,
not the grant.
FAST looks beyond individual projects to understand what is preventing promising discoveries from moving forward, whether that is missing evidence, a research resource no single lab can build alone, a gap in funding, or a development path that does not yet exist.
Then, we help build what is needed to overcome that barrier.
That approach shapes every research investment FAST makes.
Advancing Hope, Driving Discovery
Translating science into life-changing therapies
At FAST, our mission is simple yet urgent: to accelerate meaningful therapies and, ultimately, a cure for Angelman syndrome. We believe in harnessing scientific rigor, community strength, and bold partnerships to transform research into real-life outcomes for families. With every grant, collaboration, and trial, we move closer to turning hope into impact.
people worldwide estimated to be living with AS
distinct therapeutic programs initiated
invested since 2011
Looking into the future
People living with Angelman syndrome share a mutation in the 15th chromosome. People with these chromosomal changes do not produce any, or remotely enough, of something called UBE3A—which is a protein involved in brain functioning that helps us walk, talk and perform many other everyday tasks.
As a result, children and adults with AS typically have balance issues, motor impairments and can have debilitating seizures. Disruptive sleep is often a serious challenge. Some never walk. Most do not speak. Individuals with AS have a normal life expectancy, but require continuous care and are unable to live independently.
This is life today for people living with Angelman syndrome. But it won’t be for long.
Scientists believe that AS is now the neurogenetic disorder with the greatest potential to be cured. FAST is the reason why, and the force making it happen.
FAST was founded by a group of determined parents
Investments through 2024 in research grants and mission-aligned investments that advance discovery, development, and clinical enablement.
FAST invests 92% of every donation directly towards research and programs.
Each year, hundreds of volunteers show up with their time and talent to help us raise funds and awareness towards our mission.
What is Angelman syndrome?
Angelman syndrome (AS) is a rare neurogenetic disorder that is estimated to impact about one in 15,000 people, or approximately 500,000 individuals worldwide.
What Makes Our Research Model Different
We fund the critical steps between discovery and development
Promising discoveries often need additional testing and validation before they are ready for therapeutic development. FAST funds that work, helping researchers build the evidence needed to decide what should move forward.
We build the resources researchers cannot build alone
Some of the resources needed to advance Angelman syndrome research are too complex or costly for a single laboratory to create on its own. FAST helps develop shared tools and resources that researchers can use across studies, giving the research community a stronger foundation for evaluating findings and determining what should move forward.
We invest where traditional funding falls short
Promising research does not always fit the priorities or timelines of government grants and industry investment. FAST steps in when support is missing, helping researchers answer critical questions and generate the evidence needed to move an approach forward.
When an established development path does not exist, FAST may help create one. In 2024, FAST established AS²Bio, a drug development accelerator designed to advance promising science through venture philanthropy.
We bring the right expertise together
Complex research challenges rarely sit within one area of expertise. FAST brings together leaders across science, medicine, and industry to examine important questions from multiple perspectives and make stronger decisions about what should move forward.
Bringing those perspectives together helps FAST make stronger research decisions and connect promising approaches with the expertise they need to advance.
We fund the critical steps between discovery and development
Promising discoveries often need additional testing and validation before they are ready for therapeutic development. FAST funds that work, helping researchers build the evidence needed to decide what should move forward.
A Model Built for Impact
invested in research and mission-aligned initiatives
FAST’s Research Model
FAST advances Angelman syndrome research by funding critical gaps, building shared tools, and accelerating promising science toward treatment.
How FAST Advances Angelman Syndrome
What are the symptoms of
Angelman syndrome?
Delays in development
Limited or no verbal speech
Balance and coordination difficulties
Unique behaviors
Seizures
Sleep difficulties
What are the types of Angelman Syndrome?
There are multiple different mechanisms or genetic differences that can result in the absence of UBE3A protein. In AS, we call these different genetic mechanisms “genotypes.” Knowing your loved one’s AS genotype is important. While each individual living with AS is unique, individuals living with the same genotype may have certain characteristics in common.
Each genotype has a different chance to be inherited. Finally, many clinical trials have genotype in their inclusion criteria for enrollment.
Neurotypical*
Deletion
Mutation
Uniparental Disomy (UPD)
Imprinting Center Defect (ICD)
Mosaic
Life Expectancy of Angelman Syndrome
Many individuals living with Angelman syndrome have a typical lifespan, but there are factors associated with Angelman syndrome that could potentially impact a person’s lifespan. Angelman Syndrome Life Expectancy has more information about our current understanding of life expectancy in AS.
Is there a cure or treatment for Angelman Syndrome?
Currently, treatments for Angelman syndrome are generally targeted to the symptoms each individual is experiencing. For example, individuals experiencing seizures should receive treatment(s) directed at reducing or eliminating the seizures. In 2022, a consensus statement on care in AS was published, offering valuable guidance to health care providers about managing symptoms and preventative care for individuals living with Angelman syndrome.
There is currently no cure for Angelman syndrome. However, research is actively progressing, aimed at developing potential therapeutics to improve symptoms and addressing the underlying cause of AS, the missing UBE3A protein in the brain. The Drug Development Pipeline provides information on potential therapeutics that are still in early stage development as well as current ongoing clinical trials.
Research, drug development, and clinical trials
Option 3: Research model or Translational research
At the core of FAST’s mission is a commitment to bring scientific and therapeutic advancements from benchside to bedside, as quickly and safely as possible. Learn more about FAST’s current grants and research priorities
Option 4: Clinical Trial Finder or Trials & Studies
Interested in finding clinical trials and observational studies? Clinical trials are research studies designed to test whether a specific potential treatment or device is safe and effective at treating a condition or symptom.
Option 1
At FAST, we know we need as many therapeutic options as possible. To make it easier to understand how we think about therapeutic development, we’ve developed the Roadmap to a Cure, as a way to think about the different potential approaches to treating AS.
Option 2
Want to learn more about the development of potential treatments for Angelman syndrome? The Drug Development Pipeline is an interactive tool that can be used to see an overview of potential Angelman syndrome therapeutics, or enable a deep dive into a specific program or clinical trial.
Resources for Angelman Syndrome
Meet with Niki
Questions about AS genetic testing or a test report? Wondering about chances for your family? Interested in understanding your loved one’s genotype and what it means for clinical trials? Schedule a free conversation with FAST’s certified genetic counselor.
Meet with Niki
New to Angelman syndrome or to FAST? If you are new to the diagnosis of Angelman syndrome, there are two things you should know—you are not alone and THERE IS HOPE. Register with our New to FAST program to receive resources and connections. FAST is a big community, here to surround you with love, support, and strength.
Meet with Niki
There are so many ways to support FAST and our mission to —whether it’s your time, talent, or treasure, we encourage you to join in a way that inspires you.
Meet with Niki
Join our community and learn all about current clinical trials and upcoming potential therapeutics as FAST hosts the biggest science and drug development symposium every November. Or make new friends and support a local event for a great cause at a CAN fundraiser. Learn about upcoming FAST events.
Continue Exploring FAST Research
Translational Research →
Explore the science, tools, and research infrastructure helping advance Angelman syndrome research.
Drug Development →
Learn about the Angelman syndrome therapeutic pipeline, development landscape, and roadmap to future treatments.
Resources & Publications →
Browse publications, educational tools, bioresources, and funding opportunities for researchers and collaborators.
Frequently Asked Questions
AS - Does AS run in families?
Most of the time, no. Angelman syndrome is most often caused by a brand new genetic change that happened prior to conception and is not inherited from a parent. However, some genotypes have a higher chance to run in the family and testing the parents may be recommended. If you have questions about whether AS runs in your family, reach out to FAST’s genetic counselor
AS - How is Angelman syndrome diagnosed?
Currently, the diagnosis of Angelman syndrome is made based using genetic testing. Genetic testing for AS can be complicated–it typically requires more than one test to confirm the genotype or to rule out the condition. Learn more about genetic testing in Angelman syndrome
Do the symptoms or severity of Angelman syndrome vary from person to person?
Yes. Every person living with Angelman syndrome is unique in what symptoms they experience and in how they learn and develop. Knowing the genotype can provide some information on what to expect, but even within a genotype, individuals can vary in their abilities and medical issues.
For example, individuals living with Deletion AS have the highest chance to have seizures. However, even within Deletion AS, some individuals may never have a seizure, some individuals may only have a seizure once with a fever, some individuals have frequent seizures that are well-controlled with medicines, and some individuals have difficult to control seizures.
How is FAST working towards a brighter future for Angelman syndrome?
What medical care do people living with Angelman syndrome need?
FAST - How can I get involved to support FAST’s mission?
There are so many ways to lean into this effort—whether it’s your time, talent, or treasure, we encourage you to join in a way that inspires you.
- Advocate
- Learn
- Participate
- Fundraise
- Influence
Are there efforts to develop treatments for Angelman syndrome?
Yes! There are ongoing clinical trials to test the safety and effectiveness of several different potential treatments for AS happening right now. There are also several other potential therapy approaches that are in earlier stages of development, meaning that they are still being tested in cells and animal models. Visit the Drug Development Pipeline to learn more.
In AS, the unique nature of the UBE3A gene offers a promising opportunity for potential therapies. Due to UBE3A’s imprinting, individuals living with AS usually have a functional paternal copy of the UBE3A gene. This presents an exciting possibility: the paternal UBE3A gene could potentially be “turned on” to enable the production of UBE3A protein. As a result, there are therapeutic approaches that could address the underlying cause of AS that may not be possible in other similar genetic conditions. Traditional strategies used in genetic disorders, such as gene replacement therapy or enzyme replacement therapy, remain potential therapeutic options as well.
Latest News
- Industry
On October 8, 2026, ASF and FAST hosted a webinar with Ultragenyx after the announcement that their phase 3 trial for GTX-102 (aspire) did not meet primary or key secondary
- Fundraising
Before Sonia and her son José set off on Peru’s Inca Trail this September, their guides gave the group two goals: survive first, and finish if you can. For Sonia
- Industry
Read the press release
- Research
At INSYNC-AS 2026, researchers came together to examine how genes beyond UBE3A and differences within UBE3A itself may shape Angelman syndrome and future therapeutic development. Why can Angelman syndrome (AS)
- Industry
Take Action
Fundraise
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Advocate
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Participate in Research
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This is a good place to start.
Stay Connected
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Join the Community
The FAST community is incredible. We rally around each other through the ups and downs of the Angelman syndrome journey, while fighting and advocating for our loved ones living with Angelman syndrome.
Meet the community to find answers, support, resources, and more from trusted information and from people who walk in the same shoes as you.
A Message from the Community.
Hear from families who have been in your shoes and what their advice is to you.
Find an Event
Join an in-person event near you!
Request Educational Resources
Help spread education and awareness within your family and local community. From
sharing your diagnosis to educating schools, therapists, and clinicians, our resources
can help build knowledge for those in your loved one’s life.
Questions about Clinical Trials?
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Moments of Hope by a
Team Who Gets It
Ready to go? Get Involved today.
Become an Advocate
FAST and the Angelman Syndrome Foundation (ASF) have partnered to strengthen grassroots advocacy efforts across the Angelman syndrome community. Together, they launched Hope in Action: Progress through Advocacy to mobilize families and supporters around a shared mission. This initiative encourages building relationships with state, local, and federal policymakers to advance meaningful policy change. By using their stories, FAST and ASF aim to amplify the community’s voice and drive progress that improves lives—both with work depending on growing our network.
We need more advocates to keep forward, get involved, and help drive change for every individual and family impacted by Angelman syndrome.
Participate in Research
Have questions? Schedule with Niki, our genetic counselor who specializes in AS
Fuel our Pipeline
In rare diseases, the playbook is different. We’re not just funding research; we’re building the pipeline ourselves. This is patient-driven progress, and philanthropy is what powers it. Every single dollar raised has purpose and impact. These dollars move potential or investigational Angelman syndrome treatments closer to reality.
FAST offers multiple ways to give and raise funds to support our mission. Find your way!
Program History
In early 2012, FAST began funding Scott Dindot’s laboratory at Texas A&M University to understand the mechanism by which the UBE3A-antisense transcript (UBE3A-AS) silences the paternal UBE3A allele, why it is only silenced in neurons, and how that process may be leveraged as a potential therapy for AS. In late 2017, the Dindot laboratory identified an investigational ASO that, through preliminary studies, was shown to interfere with the imprinting mechanism of the paternal UBE3A allele in neurons, and unsilenced the gene. FAST wanted to ensure that the development of this program moved forward in the most expeditious, safe, and detailed manner possible, with the primary goal of moving into a human clinical trial if preclinical studies supported further investigation. To do this, FAST formed GeneTx Biotherapeutics LLC (GeneTx), a for-profit limited liability company, singularly focused to develop an investigational ASO for potential future use in a Phase 1/2 clinical trial for individuals living with AS.
Key Researchers:
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Timeline:
2012
FAST began funding Scott Dindot’s laboratory at Texas A&M University to understand the mechanism by which the UBE3A-antisense transcript (UBE3A-AS) silences the paternal UBE3A allele, why it is only silenced in neurons, and how that process may be leveraged as a potential therapy for AS.
2016
FAST created a for-profit company called GeneTx Biotherapeutics LLC specifically for one purpose: to accelerate the development of an investigational antisense oligonucleotide drug for the potential treatment of AS.
2020
The clinical trial was initiated, making this FAST-backed program the first potential disease-modifying therapy in human clinical trials for Angelman syndrome.
2022
FAST announced that the company Ultragenyx Pharmaceuticals had exercised its option and closed on its acquisition of GeneTx Biotherapeutics LLC, a biotechnology company founded by FAST.