Biblio
Generation of an induced pluripotent stem cell line (TRNDi005-A) from a Mucopolysaccharidosis Type IVA (MPS IVA) patient carrying compound heterozygous p.R61W and p.WT405del mutations in the GALNS gene. Stem Cell Res. 2019;36:101408.
. Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome. Drug Des Devel Ther. 2015;9:1937-1953.
Therapies of mucopolysaccharidosis IVA (Morquio A syndrome). Expert Opin Orphan Drugs. 2013;1(10):805-818.