Results 301 to 310 of about 249,219 (350)

Successful Long-Term Enzyme Replacement Therapy in a Patient with Delayed-Onset ADA Deficiency. [PDF]

J Clin Immunol
Toskov V, Bali P, Hershfield MS, Ehl S, Speckmann C.   +4 more
europepmc   +1 more source

Two decades of experience of the Fabry Outcome Survey provides further confirmation of the long-term effectiveness of agalsidase alfa enzyme replacement therapy. [PDF]

Mol Genet Metab Rep
Ramaswami U, Pintos-Morell G, Kampmann C, Nicholls K, Niu DM, Reisin R, West ML, Anagnostopoulou C, Botha J, Jazukeviciene D, Schenk J, Hughes DA, Giugliani R.   +12 more
europepmc   +1 more source

Effectiveness and safety of enzyme replacement therapy in the treatment of Fabry disease: a Chinese monocentric real-world study. [PDF]

Orphanet J Rare Dis
Liu Y, Li Y, Li P, Zhang S, Zhiqing Z.
europepmc   +1 more source

Multi-Year Registry Study of Elapegademase Treatment in Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Requiring Enzyme Replacement Therapy. [PDF]

J Clin Immunol
Dorsey MJ, Butte MJ, Lieberman JA, Lehman H, Fausnight T, Keller MD, Fradette C, Hershfield MS, Pozos TC, Rozova A, Wall LA, Bednarski JJ, Tarrant TK, Chong HJ, Geng B, Temin NT, Laubach SS, Lin L, Mousallem T, Walter JE.   +19 more
europepmc   +1 more source

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Enzyme replacement therapy

Trends in Biochemical Sciences, 1981
ABSTRACT: Of the many genetic diseases which are now known to be due to an enzyme deficiency, the glycolipid storage diseases are among those which are most likely to yield to enzyme replacement therapy. To be successful, such therapy requires the availability of relatively pure enzyme from a human source, delivery of the enzyme to the storage cell ...
George L. Dale, Ernest Beutler
openaire   +3 more sources

Pancreatic enzyme replacement therapy

Current Gastroenterology Reports, 2001
Malabsorption due to severe pancreatic exocrine insufficiency is one of the most important late features of chronic pancreatitis. Generally, steatorrhea is more severe and occurs several years prior to malabsorption of other nutrients because synthesis and secretion of lipase are impaired more rapidly, its intraluminal survival is shorter, and the lack
Peter Layer, Paul Georg Lankisch, Jutta Keller   +2 more
openaire   +3 more sources

Enzyme replacement therapy in Fabry disease [PDF]

Journal of Inherited Metabolic Disease, 2001
AbstractRecent clinical trials have demonstrated that enzyme replacement therapy with α‐galactosidase A (α‐Gal A) constitutes a major clinical advance in the treatment of patients with Fabry disease. This new therapeutic approach has been shown to be well tolerated and effective in reducing levels of the storage product globo‐triaosylceramide and in ...
Roscoe O. Brady, Raphael Schiffmann, Gary J. Murray, David F. Moore   +3 more
openaire   +2 more sources

Enzyme Replacement Therapy in a Gaucher Family

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.
Ilhami Berber, Neslihan Erdem, Ismet Aydogdu, Tahir Buran   +3 more
openaire   +4 more sources

Enzyme-replacement therapy: Problems and prospects [PDF]

Pharmaceutisch Weekblad Scientific Edition, 1989
Several diseases can, at least in theory, be treated by the administration of an enzyme, the deficiency of which is the cause of the disease. Various attempts have been made to correct enzymatic deficiencies responsible for the clinical manifestation of diseases for which prevention cannot be achieved by modification of the diet or by supportive ...
J. Raber, B. Rademaker
openaire   +2 more sources