Could you represent the rare experience on this new venture from The Patient Association?
Genomics England have released findings that show the public are generally supportive of using whole genome sequencing for newborn screening.
The top eleven questions that patients, families and healthcare professionals want to be addressed in liver storage disease by researchers.
Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX053, an mRNA for the Treatment of GSD3
The first patient has been dosed in the FORTIS Phase 1/2 trial of AT845, a single-dose gene therapy for adults with late-onset Pompe disease (LOPD)
Clinical Survey Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With GSD3.