Results 111 to 120 of about 1,596 (158)

Integrase-Deficient Lentiviral Vector as a Platform for Efficient CRISPR/Cas9-Mediated Gene Editing for Mucopolysaccharidosis IVA. [PDF]

Int J Mol Sci
Nidhi F, Tomatsu S.
europepmc   +1 more source

Long-term outcomes of enzyme replacement therapy from a large cohort of Korean patients with mucopolysaccharidosis IVA (Morquio A syndrome). [PDF]

Mol Genet Metab Rep
Sung J, Kim I, Im M, Ahn YJ, Kim SM, Jang JH, Park HD, Jeon TY, Ko KR, Park SJ, Lee JH, Kim EY, Cheon CK, Kang E, Moon JE, Sohn YB, Lin HY, Chuang CK, Lin SP, Cho SY.   +19 more
europepmc   +1 more source

Lentiviral Vector-Mediated <i>Ex Vivo</i> Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model. [PDF]

Hum Gene Ther
Celik B, Rintz E, Sansanwal N, Khan S, Bigger B, Tomatsu S.   +5 more
europepmc   +1 more source

Analysis of endocrine hormone metabolism level in a Chinese patient with mucopolysaccharidosis IVA: A case report. [PDF]

Medicine (Baltimore), 2018
Xu L, Ren Y, Yin J, Yang J, Liu Y, Zhang J, Zhang Y, Xiang C, Yang L.   +8 more
europepmc   +1 more source

Cardiac features and effects of enzyme replacement therapy in Taiwanese patients with Mucopolysaccharidosis IVA. [PDF]

Orphanet J Rare Dis, 2018
Lin HY, Chen MR, Lin SM, Hung CL, Niu DM, Chuang CK, Lin SP.   +6 more
europepmc   +1 more source

Clinical and molecular characteristics of colombian patients with mucopolysaccharidosis IVA, and description of a new galns gene mutation. [PDF]

Mol Genet Metab Rep, 2018
Moreno Giraldo LJ, Escudero Rodríguez ÁM, Sánchez Gómez A, Satizabal Soto JM.   +3 more
europepmc   +1 more source

Mucopolysaccharidosis type IVA and severe hidradenitis suppurativa: A case series

JAAD Case Reports
Fialová, Jorga, Kojanová, Martina, Šáhó, Robert, Magner, Martin   +3 more
openaire   +5 more sources

Muskoskeletal manifestations of mild form of mucopolysaccharidosis iva - a clinical case [PDF]

Pediatr Rheumatol Online J, 2014
Ganeva M, Stefanov S, Avdjieva D, Tincheva R, Sinigerska I, Zahariev D, Bachvarova J, Todorova Z.   +7 more
europepmc   +1 more source

Growth Patterns in MPS IVA and MPS IIIA: A Longitudinal Single-Center Study. [PDF]

J Clin Med
Carmon L, Nassar M, Idan D, Leifman D, Shaki D, Elamour S, Hershkovitz E, Loewenthal N, Haim A, Chacham OS.   +9 more
europepmc   +1 more source

Clinical and Molecular Characterization of Pakistani Mucopolysaccharidosis Families with <i>SGSH</i> and <i>GALNS</i> Deficiencies. [PDF]

Genes (Basel)
Awan FN, Zulfiqar S, Eiman L, Asif M, Hussain MS, Dahl N, Baig SM, Oda H.   +7 more
europepmc   +1 more source