Ultragenyx Pharmaceutical (RARE) shows promising results for GTX-102 in Angelman syndrome Phase 1/2 trial, supporting a Phase ...

Tulare County Sheriff Mike Boudreaux awarded the Medal of Valor to Deputy Alfredo Flores on Nov. 12 for rescuing Angel TaFoya ...

Ultragenyx Pharmaceutical Inc. (RARE) announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its ...

Phase 1/2 data show improvements across all domains and confirm that Phase 3 Aspire study is amply powered to establish efficacy of GTX-102Phase ...

Munchausen syndrome is a factitious disorder, a mental health disorder in which a person repeatedly and deliberately acts as if they have a physical or mental illness when they are not really sick.

New preclinical data for candidate ETX201 illustrate its potential as a first-in-class gene therapy for Angelman syndrome. Studies in non-human primates demonstrate safety, biodistribution ...

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced Saturday that GTX-102, its candidate for the rare neuro-genetic disorder Angelman syndrome, improved patient outcomes across multiple domains in a ...

The official condition is called brachycephalic obstructive airway syndrome (BOAS), which will limit the dog’s ability to ...

Tulare County Sheriff’s Deputy Alfredo Flores was awarded the Medal of Valor Tuesday. He rescued 12-year-old Angel from a ...

(NASDAQ: RARE) today announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, that will be presented at the ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) has confirmed plans to initiate a Phase 3 study for ION582, a potential treatment for Angelman syndrome (AS), in the first half of 2025. The decision ...

for RMB 100 million yuan, providing a treatment option for deficiency syndrome and has been included in treatment protocols in multiple ... Additionally, the university has established partnerships ...