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 ...

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 ...

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 ...

Also known as Selective Sound Sensitivity Syndrome (4S), it applies to patients who have aversion to very specific sounds, such as chewing, breathing, click pen, snapping lips, wheezing etc. 1 - 6 ...