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

NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug ...

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 ...

All told, 97% of people in the medium and high dose groups saw an improvement in overall symptoms measured using the Symptoms of Angelman Syndrome–Clinician Global Impression-Change (SAS-CGI-C ...

Some people with Angelman syndrome have a “misprint” in the gene. Others are missing that chapter entirely. Every cell has two copies of each gene, one from each parent.

"Dosing the first person with Angelman syndrome in our pivotal REVEAL study marks an important milestone for this underserved community, who currently have no approved disease modifying treatments ...

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 ...

Angelman syndrome is a rare neurodevelopmental disorder arising primarily from loss of function of the maternally inherited UBE3A gene. Research has shown that UBE3A plays a crucial role in ...

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 ...