An Intravenous Brain-Penetrant Enzyme Therapy for Mucopolysaccharidosis II.
Muenzer J, Burton BK, Harmatz P, Rajan DS, Jones SA, van den Hout JMP, Mitchell JJ, Bhalla A, Engmann NJ, Zubizarreta I, Dong W, Model F, Watts RJ, Troyer MD, Chin PS, Ho C.
Muenzer J, et al. Among authors: ho c.
N Engl J Med. 2026 Jan 1;394(1):39-50. doi: 10.1056/NEJMoa2508681.
N Engl J Med. 2026.
PMID: 41467650
Clinical Trial.