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

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 ...

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 ...