Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide ...

Angelman syndrome is caused by a large deletion of the region of the maternal chromosome that contains UBE3A, or by a DNA sequence change (mutation) in the UBE3A gene inherited from the mother.

is a disorder of the nervous system characterized by developmental disabilities, seizures, speech deficits, and motor oddities. The disease is named after English physician Harry Angelman ...

Alström Angels is a 501 (c)(3) non-profit charity dedicated to raising funds for Alström Syndrome genetic and medical research, increasing awareness of the rare disease. Purchase tickets and ...