A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B.
Muschol N, Koehn A, von Cossel K, Okur I, Ezgu F, Harmatz P, de Castro Lopez MJ, Couce ML, Lin SP, Batzios S, Cleary M, Solano M, Nestrasil I, Kaufman B, Shaywitz AJ, Maricich SM, Kuca B, Kovalchin J, Zanelli E.
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J Clin Invest. 2023 Jan 17;133(2):e165076. doi: 10.1172/JCI165076.
J Clin Invest. 2023.
PMID: 36413418
Free PMC article.
Clinical Trial.