Gene Therapy for HD Gets FDA Orphan Designation – Rare Disease Report
Rare Disease Report |
Gene Therapy for HD Gets FDA Orphan Designation
Rare Disease Report uniQure N.V., the company that developed the first-ever approved gene therapy (for familial chylomicronemia syndrome), announced that the U.S. Food and Drug Administration (FDA) has now granted an Orphan Drug Designation for its gene therapy … uniQure Announces FDA Orphan Drug Designation for AMT-130 in Huntington's disease~ First Investigational Gene … uniQure (QURE) Granted FDA Orphan Drug Designation for AMT-130 in Huntington's Disease |
