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

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 ...

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

The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system (CNS), a naturally occurring phenomenon in which the ...

Angelman syndrome is a rare condition that causes problems with growth and development. It’s caused by a genetic mutation (a change in your genes) that affects the nervous system. It’s named ...

The initial participant in the pivotal Phase 3 REVEAL trial, which is assessing ION582 for Angelman syndrome, a severe neurodevelopmental disease, has been dosed by Ionis Pharmaceuticals, Inc ...

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 ...

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the first participant has been dosed in the global Phase 3 REVEAL study, which is designed to evaluate the efficacy and safety of ...

Ionis Pharmaceuticals has dosed the first subject in the global, randomised Phase III REVEAL trial of investigational ION582, aimed at treating individuals with Angelman syndrome (AS), a rare ...

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

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.