For the first time, Colin Farrell has opened up his home and talked in-depth about life with his son James, who has Angelman ...

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

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced Saturday that GTX-102, its candidate for the rare neuro-genetic disorder ...

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

Colin Farrell’s son James had a part to play in his sobriety journey. The now-21-year-old has Angelman syndrome, and the actor called his child’s diagnosis “fuel” in choosing to abstain fr ...

Joseph Schwartz has given his Buy rating due to a combination of factors surrounding Ultragenyx Pharmaceutical’s GTX-102 program for Angelman syndrome. The updated data from the Phase 1/2 trial, ...

A four-year-old girl with DUP15q syndrome, who doctors said would never walk, has defied expectations to take her first steps.

A four-year-old girl who doctors said would never walk has defied the odds and taken her first steps. Scottie Mae Blair was ...

Ionis has announced the design for a Phase III trial of ION582, a treatment for Angelman syndrome (AS), following discussions with the FDA.

THEIR KCCI PRODUCER, HOPE EALING’S FATHER AND BROTHER BRANDON WAS DIAGNOSED WITH ANGELMAN SYNDROME WHEN HE WAS A LITTLE LITTLE, WHICH IMPEDES HIS ABILITY TO WALK, LET ALONE RUN. THEY COMPLETED ...

Overall, 97% of people in the medium and high dose groups assessed in the study saw an improvement in overall AS symptoms as measured by the Angelman Syndrome Clinical Global Improvement Change ...

A four-year-old girl who doctors said would never walk has defied the odds and taken her first steps. Scottie Mae Blair was ...