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 ...

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 ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) is among the 11 Best Genomics Stocks to Buy According to Hedge Funds. The initial participant in the pivotal Phase 3 REVEAL trial, which is assessing ...

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 ...

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 ...

Seventeen-year-old Lucas, a cheerful and affectionate boy from Singapore, lives with Angelman syndrome (AS), a rare neurogenetic condition that affects roughly 1 in every 15,000 live births.

In moderate to severe cases, people experience restless legs syndrome several times a week, and in the most extreme cases, symptoms can delay sleep for several hours, said Dr. Brian Koo, associate ...

Two strong components play a role in who gets restless legs syndrome: genetics and iron levels. Restless legs syndrome often runs in families, and genetic markers make up about 20% of the ...