Results 101 to 110 of about 23,853 (147)

Safety and efficacy of laronidase in Chinese patients with mucopolysaccharidosis type I: a phase IV, single-arm, open-label, multicenter study. [PDF]

Orphanet J Rare Dis
Liang Y, Yang YL, Zou CC, Liu L, Jiao Y, Wang Y, Liu X, Luo XP.   +7 more
europepmc   +1 more source

Expanding the Spectrum: A Rare Case of Morquio Syndrome With Bronchial Asthma and Seizure Disorder. [PDF]

Clin Case Rep
Bastola B, Dahal S, Acharya RR, Sah M, Wagle P.   +4 more
europepmc   +1 more source

Allogeneic hematopoietic stem cell transplantation modulates neurodevelopmental trajectories in mucopolysaccharidosis: a longitudinal study of subtype-specific outcomes and age-dependent efficacy. [PDF]

Orphanet J Rare Dis
Xu Y, Fang X, Luo C, Zhou C, Wang J, Mi Y, Xie J, Shen M.   +7 more
europepmc   +1 more source

Case report: Successful treatment of a patient presenting with a very rare association of acute lymphoblastic leukemia and mucopolysaccharidosis type IVA. [PDF]

Front Pediatr
Arnaboldi SMC, Faraguna MC, Colombini A, Sala A, Leoni V, Spinelli M, Gotti G, Bettini LR, Crescitelli V, Commone A, Gasperini S, Rizzari C.   +11 more
europepmc   +1 more source

Challenges and opportunities with providing genetic testing and counseling for mucopolysaccharidosis type II in Kenya. [PDF]

Orphanet J Rare Dis
Mungai LNW, Njeru C, Njoroge A, Maina M, Ilovi S, Nduati RW, Wamalwa D, Odongkara B, Miller DE.   +8 more
europepmc   +1 more source

Children and Adolescents with Mucopolysaccharidosis and Osteogenesis Imperfecta: The Dentistry on the Multiprofessional Team. [PDF]

J Pers Med
Celestino MLS, Carneiro NCR, Prado HV, Cabral GMP, Abreu MHNG, Borges-Oliveira AC.   +5 more
europepmc   +1 more source

Mucopolysaccharidosis type IVA and severe hidradenitis suppurativa: A case series. [PDF]

JAAD Case Rep
Fialová J, Kojanová M, Šáhó R, Magner M.   +3 more
europepmc   +1 more source

Adeno-associated vector corneal gene therapy reverses corneal clouding in a feline model of mucopolysaccharidosis VI. [PDF]

PLoS One
Gilger BC, Song L, Hasegawa T, Salmon JH, Bower JJ, Vridhachalam N, Collins E, Roberts D, Bagel J, Molony C, Vite C, Hirsch ML.   +11 more
europepmc   +1 more source

The use of genistein and ambroxol may be an effective approach in correcting cellular dysfunctions of mucopolysaccharidosis-plus syndrome. [PDF]

Mamm Genome
Cyske Z, Rintz E, Gaffke L, Pierzynowska K, Węgrzyn G.   +4 more
europepmc   +1 more source