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 ...

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 ...

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 ...

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) is among the 11 Best Genomics Stocks to Buy According to Hedge Funds. The initial ...

CARLSBAD, Calif., June 11, 2025--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 ...

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 ...