Results 91 to 100 of about 783 (129)

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

Heparan sulfate in cerebrospinal fluid as a biomarker to assess disease severity and for treatment monitoring in patients with Mucopolysaccharidosis Type II: a position statement. [PDF]

Orphanet J Rare Dis
Giugliani R, de Siqueira ACM, Santos ES, Leão EKEA, Carvalho GDS, Santos MLSF, Raskin S, Martins AM.   +7 more
europepmc   +1 more source

Harmonizing Perspectives on MPS II Care in Türkiye: A Delphi Study Towards Treatment Management Consensus. [PDF]

Healthcare (Basel)
Mungan NO, Tumer L, Sivri S, Arslan N, Ucar SK, Seker Yilmaz B, Gokcay G.   +6 more
europepmc   +1 more source

Advances in Therapies for Mucopolysaccharidoses. [PDF]

Curr Issues Mol Biol
Szmydtka J, Ziemian M, Banaszak R, Ciesielska M, Ładosz J, Maliszewska U, Nowakowski A, Paszek M, Suproń G, Świętochowska Z, Tkaczuk A, Wojciechowski S, Węgrzyn G.   +12 more
europepmc   +1 more source

Novel Phenotypic Insights into the <i>IDS</i> c.817C>T Variant in Mucopolysaccharidosis Type II from Newborn Screening Cohorts. [PDF]

Int J Neonatal Screen
Beauregard-Lacroix É, Menello C, Steffensen M, Lin HY, Chuang CK, Lin SP, Ficicioglu C.   +6 more
europepmc   +1 more source

How to manage hypersensitivity reactions to enzyme replacement therapy in lysosomal storage diseases? [PDF]

Orphanet J Rare Dis
Spataro F, Solimando AG, Desantis V, Vacca A, Di Girolamo A, Ria R.   +5 more
europepmc   +1 more source

Advances in Therapeutic Options for Pulmonary and Sleep Disorders in Mucopolysaccharidosis (MPS) Patients: A Narrative Review. [PDF]

Adv Respir Med
Akpa B.
europepmc   +1 more source

Radiographic assessment of mucopolysaccharidoses: A pictorial review. [PDF]

World J Clin Pediatr
Teixeira de Castro Gonçalves Ortega AC, Amorim Moreira Alves G, Perera Molligoda Arachchige AS.   +2 more
europepmc   +1 more source

Designing better gene therapies for lysosomal storage disorders using engineered enzymes. [PDF]

Mol Ther
Miller-Hodges E, Bigger B.
europepmc   +1 more source

Characterization of Treatment Utilization in a Metabolic Genetics Clinic. [PDF]

J Pediatr Pharmacol Ther
Riedy M, Briggs J, Huang T.
europepmc   +1 more source