Biblio
Therapies of mucopolysaccharidosis IVA (Morquio A syndrome). Expert Opin Orphan Drugs. 2013;1(10):805-818.
Molecular Genetics and Metabolism Report Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: clinical, biochemical, and pathological improvements. Mol Genet Metab Rep. 2015;2:65-76.
Impact of Enzyme Replacement Therapy and Hematopoietic Stem Cell Therapy on Growth in Patients with Hunter Syndrome. Mol Genet Metab Rep. 2014;1:184-196.
Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome. Drug Des Devel Ther. 2015;9:1937-1953.
Hematopoietic stem cell transplantation for mucopolysaccharidoses; past, present, and future. Biol Blood Marrow Transplant. 2019.