Author Archives: Margarida Maia PhD

FAST Science Summit Brings Message of Hope and Raises Funds

Researchers brought a message of hope for the Angelman syndrome community as they discussed advances in treatment and scientific knowledge at the recent 2021 FAST Global Science Summit and Gala in Austin. Hosted by the Foundation for Angelman Syndrome Therapeutics (FAST), the annual event is an opportunity for members…

Nonprofits Seek to Add Angelman to Newborn Screening in N. Carolina

Four foundations are collaborating to fund the addition of Angelman syndrome and other conditions caused by changes on chromosome 15 to Early Check, a free, voluntary study that screens newborn babies in North Carolina for rare diseases. These foundations include the Foundation for Angelman Syndrome Therapeutics (FAST), the Angelman…

FDA Clears Way for KIK-AS Phase 1/2 Trial to Resume

The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on a Phase 1/2 study testing GTX-102 — an investigational therapy by GeneTx Biotherapeutics and Ultragenyx Pharmaceutical — in children and adolescents with Angelman syndrome. GeneTx is now cleared to begin dosing treatment-naïve patients — those who have never received…