Results 131 to 140 of about 1,275 (171)

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

BioDrugs
Zanetti A, Tomanin R.
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

The top 100 most cited articles on mucopolysaccharidoses: a bibliometric analysis. [PDF]

Front Genet
Liao R, Geng R, Yang Y, Xue Y, Chen L, Chen L.   +5 more
europepmc   +1 more source

Access to drugs for rare diseases (DRD) in Canada: a comprehensive review of the provincial DRD-specific programs. [PDF]

Ther Adv Rare Dis
Awada N.
europepmc   +1 more source

Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan. [PDF]

Orphanet J Rare Dis
Nakamura K, Sakai N, Hossain MA, Eisengart JB, Yamamoto T, Tanizawa K, So S, Schmidt M, Sato Y.   +8 more
europepmc   +1 more source

Respiratory insufficiency after brain metastasectomy for extraskeletal Ewing sarcoma in an adult patient with mucopolysaccharidosis type II: a case report. [PDF]

Int Cancer Conf J
Hoshi M, Ban Y, Iwai T, Takada N, Oebisu N.   +4 more
europepmc   +1 more source

Caring for Patients with Hunter Syndrome [PDF]

, 2014
Teitsma, Nicole
core   +1 more source