Therapy Targeting CF Mutation Appears to Restore CFTR Gene in Homozygous Patients in Study
ProQR Therapeutics is reporting that an early clinical study of QR-010 in cystic fibrosis patients homozygous for the ΔF508 mutation in the CFTR gene met its primary endpoint. After four weeks of treatment, these patients showed a positive change in total chloride response, meaning that the CFTR protein was again working. In patients heterozygous for…