NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has ...

Prader-Willi syndrome is a study in contrast — children who once couldn’t eat later battle an urge to never stop. The ...

Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif., June 27 ...

Ultragenyx Pharmaceutical received breakthrough therapy designation from the U.S. Food and Drug Administration for its drug GTX-102, which is designed to treat people with Angelman syndrome. Angelman ...

In a conversation with People magazine in 2024, Colin Farrell opened up for the first time about his son's rare condition: ...

More information: Jacqueline Fátima Martins de Almeida et al, Molecular aspects of Angelman Syndrome: Defining the new path forward, Biomolecules and Biomedicine (2025). DOI: 10.17305/bb.2025.11724 ...

It is caused by the loss of function of the UBE3A gene in neurons. Angelman syndrome is thought to affect 1:12,000-1:20:000 individuals. There are currently no approved treatments for Angelman ...

MIDDLETON, Mass., May 12, 2025 /PRNewswire/ -- MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today ...

Angelman syndrome is thought to affect 1:12,000-1:20:000 individuals. There are currently no approved treatments for Angelman syndrome. About MVX-220 ...

In Angelman syndrome, only the copy of the UBE3A gene inherited from the mother has a genetic variant (either a mutation or deletion), while the father’s allele is silenced through the imprinting ...

Angelman Syndrome (AS) is a rare genetic disorder that primarily affects neurological development, characterized by developmental delays, intellectual disability, speech impairment, and movement ...