Results 271 to 280 of about 460,260 (336)

Real-life impacts of olipudase alfa: experiences of adults receiving enzyme replacement therapy for acid sphingomyelinase deficiency-results from an international survey study. [PDF]

Orphanet J Rare Dis
Morsy AS, Mbua S, Mathieson T, Hopkin J, Bolton S.   +4 more
europepmc   +1 more source

Pancreatic Enzyme Replacement Therapy in Pancreatic Exocrine Insufficiency-Real-World's Dosing and Effectiveness: A Systematic Review. [PDF]

Dig Dis Sci
Kadaj-Lipka R, Monica M, Stożek-Tutro A, Ryś P, Rydzewska G.   +4 more
europepmc   +1 more source

Impact of enzyme replacement therapy on clinical manifestations in females with Fabry disease. [PDF]

Orphanet J Rare Dis
Lenders M, Nowak A, Cybulla M, Kaufeld J, Köhn AF, Muschol NM, Kurschat C, Brand E.   +7 more
europepmc   +1 more source

Amelioration of Inflammation and Metabolic Blockage in GALC Deficient Mice After Enzyme Replacement Therapy via Extracellular Vesicles. [PDF]

Int J Nanomedicine
Zelada D, Saldivia N, Samano S, George D, Chaudhari K, Nguyen D, Simchuk D, Van Breemen RB, Givogri MI.   +8 more
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

Case Report: A case of surgical and enzyme replacement therapy for type I Gaucher disease complicating femoral shaft pathological fracture. [PDF]

Front Surg
Gao Y, Liu J, Zhang Z, Sheng X, Wang Y, Zhao X, Ma H.   +6 more
europepmc   +1 more source

Enzyme Replacement Therapy in a Gaucher Family [PDF]

Journal of the National Medical Association, 2018
Gaucher disease is a lipid storage disorder due to deficiency of beta glucocerebrosidase. It's an autosomal recessive disease and as a result of this enzyme deficiency, glucocerebroside accumulates in various types of tissues like liver, brain spleen and bone marrow.
Neslihan, Erdem, Tahir, Buran, Ilhami, Berber, Ismet, Aydogdu   +3 more
openaire   +4 more sources

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Enzyme replacement therapy.

Survey of Ophthalmology, 2019
Table ...
A. Moore
semanticscholar   +3 more sources

Enzyme Replacement Therapy [PDF]

, 2018
Enzyme replacement therapy is a therapeutic approach in which the specific enzyme that is absent or inactive in affected individuals is replaced with a functional enzyme molecule derived from biological sources or produced by biotechnology. A large number and variety of enzyme defects have been identified in humans.
Abderrezak Khelfi
openaire   +2 more sources

Enzyme Replacement Therapy for Genetic Disorders Associated with Enzyme Deficiency.

Current Medicinal Chemistry, 2021
Mutations in human genes might lead to loss of functional proteins, causing diseases. Among these genetic disorders, a large class is associated with the deficiency in metabolic enzymes, resulting in both an increase in the concentration of substrates ...
Marialaura Marchetti, S. Faggiano, A. Mozzarelli   +2 more
semanticscholar   +1 more source