Trial for Gene Therapy to Treat Sanfilippo Begins in Spain – Rare Disease Report
Rare Disease Report |
Trial for Gene Therapy to Treat Sanfilippo Begins in Spain
Rare Disease Report We remain encouraged by the improvements observed in clinically relevant biomarkers post-dosing of the gene therapy in the patients in Cohort 3 and the ongoing safety profile ABO-102 demonstrates,” stated Juan Ruiz, M.D., Ph.D., Chief Medical Officer … |
