This trial will be testing a new drug called Alogabat that is meant to help 5-17 year olds with Angelman Syndrome.

Para nuestro proyecto, estamos estudiando una evaluación del desarrollo infantil que pueda hacerse a distancia desde la casa de la familia. Esta se llama "Evaluación del neurodesarrollo con ayuda de los padres" o "PANDABox", según su acrónimo. PANDABox se ha usado con familias que hablan inglés, pero no se había traducido al español hasta ahora. De modo que hay dos razones por las que estamos haciendo nuestro estudio: primero, traducir la evaluación al español; y segundo, probar la evaluación con familias latinas con bebés que podrían beneficiarse de una evaluación del desarrollo. Después de probar la evaluación, les preguntaremos a las familias cómo fueron sus experiencias y si tienen alguna sugerencia para mejorarla. En general, esperamos mejorar la evaluación PANDABox en cuanto al idioma y la cultura de las familias que hablan español.

This study is taking place to create a first-year-of-life, MRI-based predictive test for later autism spectrum disorder (ASD) diagnosis. Subjects will travel to UNC to be assessed using MRI as well as behavioral assessments and observations at 6, 12 and 24 months of age. We will also collect information about the subject's family to gather data on their environment.

The Registries inform individuals with autism, fragile X and intellectual and developmental disabilities about research studies.

(1) To translate a remote developmental assessment into Spanish with feedback from the Spanish-speaking community (2) To try out the assessment with Latinx infants who are showing risk signs of a developmental disability. After completing the assessment, we will interview families about their experiences. We hope that this information will help us improve remote assessments for Latinx families to better match their culture and language.

In this study we will look at sleep problems in the home as well as behaviors and differences in the brain (from infancy) associated with sleep problems in school-age. Parents of the participants of our prior study will participate by providing information on their child's sleep habits using a sleep watch, and completing a sleep diary and parent reports.

The purpose of this study is to evaluate if the drug GTX-102 is effective with the Angelman population. This is a Phase 3 study, that is a double-blind, sham-controlled study.

To evaluate the efficacy of ION582 in participants with Angelman syndrome (AS) receiving ION582 vs Placebo as measured through expressive communication as well as measured through functional domains, including overall symptoms of disease severity, cognition, communication, sleep, motor functioning, and daily living skills.