Translating a novel fetal therapy for lysosomal diseases into clinical care: The race for approval to treat one patient with mucopolysaccharidosis type VII.

Molecular genetics and metabolism
2020
https://researcherprofiles.org/profile/151837341
Marisa Eve Pulcrano, Billie Lianoglou, Juan Gonzalez-Velez, Renata C. Gallagher, Mary E. Norton, Tippi MacKenzie