Results 81 to 90 of about 172,212 (109)

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

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

Non-Invasive Device-Mediated Drug Delivery to the Brain across the Blood-Brain Barrier. [PDF]

Pharmaceutics
Tashima T, Tournier N.
europepmc   +1 more source

Case report of neuronopathic mucopolysaccharidosis type II: Early intracerebroventricular enzyme replacement therapy and hematopoietic cell transplantation with developmental outcomes up to 5 years of age. [PDF]

Mol Genet Metab Rep
Ikari A, Hama A, Okuyama T.
europepmc   +1 more source

α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial. [PDF]

Mol Ther
Harmatz P, Giugliani R, Martins AM, Hamazaki T, Kubo T, Kira R, Minami K, Ikeda T, Moriuchi H, Kawashima S, Takasao N, So S, Sonoda H, Hirato T, Tanizawa K, Schmidt M, Sato Y.   +16 more
europepmc   +1 more source

1973. Domenico Rea a Alfa Romeo - Alfa sud

, 1973
Rea, Domenico, Alfa Romeo - Alfasud
core   +1 more source

Integrative genomic and single-cell framework identifies druggable targets for colorectal cancer precision therapy. [PDF]

Front Immunol
Hong Y, Li J, Xu N, Shu W, Chen F, Mi Y, Geng H, Li Q.   +7 more
europepmc   +1 more source

Oral trehalose improves histological and behavior symptoms of mucopolysaccharidosis type II in iduronate 2-sulfatase deficient mice. [PDF]

Sci Rep
Lee H, Han JH, Jeong RG, Kang YJ, Choi BH, Kim SR, Cheon CK, Hur J, Lee SY.   +8 more
europepmc   +1 more source

Efficient brain delivery and sustained retention of antibodies via dual targeting of blood-brain barrier and intra-brain proteins. [PDF]

MAbs
Noguchi Y, Hironiwa N, Sato K, Yamazaki M, Kawashima M, Nagaya N, Nakagawa K, Okuda M, Kato A, Shu F, Haraya K, Kuramochi T.   +11 more
europepmc   +1 more source