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 ...

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.

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

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

After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...

Shaken baby syndrome (SBS) is a violent act of abuse that can cause myriad neurologic, cognitive, and other functional deficits. In the most serious cases, death can result. Health care ...

This is the heartbreaking reality for parents who experience Sudden Infant Death Syndrome (SIDS), a tragedy that strikes without warning, leaving families devastated. Dr Christine Wanjiru Njagi ...

In the less severe cases, the infant may present with the parents or caretaker relaying a history of poor feeding, vomiting, lethargy or irritability, hypothermia or chills, a generalized failure ...