Results 81 to 90 of about 550 (133)

Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder? [PDF]

Int J Mol Sci, 2022
Horgan C, Jones SA, Bigger BW, Wynn R.
europepmc   +1 more source

Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II. [PDF]

Orphanet J Rare Dis
Yee KS, Lewis S, Evans E, Romano C, Alexanderian D.   +4 more
europepmc   +1 more source

Ancestral lysosomal enzymes with increased activity harbor therapeutic potential for treatment of Hunter syndrome. [PDF]

iScience, 2021
Hendrikse NM, Sandegren A, Andersson T, Blomqvist J, Makower Å, Possner D, Su C, Thalén N, Tjernberg A, Westermark U, Rockberg J, Svensson Gelius S, Syrén PO, Nordling E.   +13 more
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

Production of active human iduronate-2-sulfatase (IDS) enzyme in Nicotiana benthamiana. [PDF]

Sci Rep
Sinha MH, Mehtab T, Entesari M, Nguyen HH, Yun A, Hwang I.   +5 more
europepmc   +1 more source

Efficacy and safety of idursulfase beta in the treatment of mucopolysaccharidosis II: A phase-3, 2-part study compared with a historical placebo cohort

Genetics in Medicine
Young Bae Sohn, Aram Yang, Min-Sun Kim, Jinsup Kim, Jae Seong Kim, Youngsin Oh, Dong-Kyu Jin   +6 more
openaire   +1 more source

Analysis of fatal outcomes of patients with mucopolysaccharidosis type II according to the Russian mucopolysaccharidosis registry. [PDF]

World J Clin Pediatr
Buchinskaya N, Vechkasova A, Vashakmadze N, Namazova-Baranova L, Ivanov D, Zakharova E, Kutsev S, Kostik M.   +7 more
europepmc   +1 more source

Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and Beyond. [PDF]

BioDrugs
Zanetti A, Tomanin R.
europepmc   +1 more source

Developing Treatments for Rare Diseases on a Shoestring. [PDF]

GEN Biotechnol, 2023
Puhl AC, Negri S, Hupcey MAZ, Ekins S.
europepmc   +1 more source

Prenatal Delivery of Enzyme Replacement Therapy to Fetuses Affected by Early-Onset Lysosomal Storage Diseases. [PDF]

Am J Med Genet C Semin Med Genet
Borges B, Canepa E, Chang IJ, Herzeg A, Lianoglou B, Kishnani PS, Harmatz P, MacKenzie TC, Cohen JL.   +8 more
europepmc   +1 more source