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 ...

Phenotype–genotype Correlations Clinical severity does depend on mutation type and position. A number of reports have appeared in recent years dealing with the relationships between the clinical ...

Alternatively, an indirect approach would be to employ available pharmacologic agents, such as the serotonin-reuptake inhibitor class of drugs, to address the core problems of anxiety and fear or ...

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 ...

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 ...

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.

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 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 ...

CARLSBAD, Calif. - Ionis Pharmaceuticals (NASDAQ:IONS), a $5.86 billion market cap biotechnology company, announced Wednesday that the first participant has been dosed in its global Phase 3 REVEAL ...