Results 141 to 150 of about 1,451,354 (224)

A review of gene therapy in canine and feline models of lysosomal storage disorders [PDF]

, 2015
Bradbury, Allison M, Casal, Margret L, Gurda, Brittney L, Haskins, Mark E, Ponder, Katherine P, Vite, Charles H   +5 more
core   +2 more sources

Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA [PDF]

, 2012
A Dorfman, A James, A Koto, A McDonald, A Sheiham, AH Yeung, AM Montaño, ATS Committee on Proficiency Standards for Clinical Pulmonary Function Laboratories, B Käsmann-Kellner, BS Danes, C. Gail Summers, CF Wippermann, Christian J. Hendriksz, Ciarán Mc Ardle, CJ Pelley, DB Davis, DM Gross, E Franco, E Schleier, ED Riedner, Elizabeth Braunlin, Elizabeth Wright, F Bromo, F Nicolini, FA Abraham, G Keleman, GL Semenza, GN Lael, H Northover, H Olsen, I Rolling, IH Maumenee, J Ashraf, J Couprie, J Glössl, J Lustmann, J Muenzer, J Nelson, J Nelson, J Nelson, J Nelson, JE Wraith, JE Wraith, JF Brailsford, JG Leroy, JH Dangel, JK Bredenkamp, JL Ashworth, JL Clausen, JP Gills, Kenneth I. Berger, KG Belani, L Geetha, LB Cantor, LS Leung, M Cahane, M Ghosh, M Iwamoto, M Sibilio, MA Gatzoulis, MA Ireland, Maisoon Al-Jawad, ME Dangel, ME Peters, MJ Kinirons, MO Barry, MR Pritzker, N Latif, P Harmatz, P Harmatz, P Harmatz, PD Robinson, PP Walker, PS Pagel, R Minami, R Minami, Rebecca Lawrence, RM John, RM Schieken, RO Bonow, RO Bonow, RT Sataloff, S Desai, S Kato, S Sato, S Tomatsu, S Tomatsu, Sara M. Hawley, SJ Swiedler, SL Shih, SM Factor, T Kuratani, T Leslie, TZ Nursal, UR Mohan, V Fesslová, W Holzgreve, WJ Buhain, WS Sly, Y Yamakoshi   +99 more
core   +2 more sources

Efficacy of different treatment strategies in patients with mucopolysaccharidosis: a systematic review and network meta-analysis of randomized controlled trials. [PDF]

Orphanet J Rare Dis
Huang L, Wu J, Tang B, Wu J, Wei F, Li HQ, Li L, Wang X, Wang B, Wu W, Hong X.   +10 more
europepmc   +1 more source

A novel image-based classification system for atlantoaxial deformity caused by mucopolysaccharidosis type IVA: an efficacy evaluation. [PDF]

J Orthop Surg Res
Zhang QQ, Wen-Ji S, Song J, Liang ZH, Zhou FC, Liu HT, Shao J, Zhang YH.   +7 more
europepmc   +1 more source

Hearing loss in patients with Morquio A syndrome: A scoping review. [PDF]

Medicine (Baltimore)
Diaz-Ordoñez L, Duque-Cordoba PA, Silva-Cuero K, Gutierrez-Medina JD, Saldarriaga W, Murgašová L, Magner M, Candelo E, Pachajoa H.   +8 more
europepmc   +1 more source

Nebulized and intravenous enzyme replacement therapy in mice with mucopolysaccharidosis type II. [PDF]

PLoS One
Shamoun AJ, Reis G, Ashley M, Labilloy A, Ferreira LF.   +4 more
europepmc   +1 more source

A Rare Case of Morquio Syndrome in Palestine: Clinical, Radiological, and Genetic Insights. [PDF]

Cureus
Farah RE, Farah RE, Ayasa IW, Abuisneina S.   +3 more
europepmc   +1 more source

Mucopolysaccharidosis Types IV and VI.

Indian pediatrics, 2002
openaire   +1 more source

Clinical, biochemical, and molecular characteristics of Sanfilippo a syndrome (MPS IIIA) in a cohort of Egyptian patients. [PDF]

Orphanet J Rare Dis
Fateen E, Nosier SS, Aziz NNA, Radwan AM, Mohammed EEA.   +4 more
europepmc   +1 more source

Challenges and solutions in the treatment of spinal disorders in patients with skeletal dysplasia: A comprehensive review. [PDF]

World J Methodol
Tsirikos AI, Jain A, Ahuja K.
europepmc   +1 more source