The first patient has been dosed in Canada as part of a Phase 1/2 clinical trial of the investigational therapy GTX-102 in…
Articles by Patricia Inacio, PhD
A professor at the University of California (UC) Davis Health has been awarded a $1.36 million Discovery Stage Research Projects…
The United Kingdom’s Medicines Healthcare Products Regulatory Agency (MHRA) has authorized a Phase 1/2 clinical trial to test the investigational therapy GTX-102 in…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
Health Canada has given the green light for a Phase 1/2 clinical trial to resume enrollment to test the investigational…
Ovid Therapeutics is stopping its clinical program for OV101 (gaboxadol), a candidate therapy for both…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BIO017, a candidate therapy for Angelman…
The Foundation for Angelman Syndrome Therapeutics (FAST) has awarded researchers with a grant to develop novel methods to accurately assess…