Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the first participant has been dosed in the global Phase 3 ...

Background/aim Prader-Willi syndrome (PWS) and Angelman syndrome (AS) are rare imprinting disorders caused by the aberrant ...

Stetson was diagnosed with Angelman syndrome a couple months later, when the results of genetic blood work revealed that he had a deleted gene on the maternally derived Chromosome 15. The neurogenetic ...

Danny and Arnie started whispering about Colin after Danny complained that he was wearing a sweater for their episode of ...

END SEPSIS proudly announces the reintroduction of the SEPSIS Act (S.1929), a bipartisan bill that marks a significant step toward creating a comprehensive national response to the sepsis crisis.

A new drama focusing on a character with autism who has a unique way of solving crimes is coming to television.

Travere Therapeutics, Inc., today announced that the Company will present three abstracts on the effect of FILSPARI (sparsentan) in rare kidney disease at the upcoming International Podocyte ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

A new specialty center opened Tuesday at the Hospital of the University of Pennsylvania in Philadelphia. It's one of the ...

The FDA denied a lifesaving drug for Barth syndrome. Now, one Colorado family fears their baby’s only treatment could be pulled.

Dawson Grant was born with several birth defects and underwent many surgeries. He is now thriving and is like "any 2-year-old ...