Results 261 to 270 of about 133,872 (296)
Some of the next articles are maybe not open access.
Enzyme replacement therapy in a mouse model of aspartylglycosaminuria
The FASEB Journal, 2000ABSTRACT Aspartylglycosaminuria (AGU), the most common lysosomal disorder of glycoprotein degradation, is caused by deficient activity of glycosylasparaginase (AGA). AGA‐deficient mice share most of the clinical, biochemical and histopathologic characteristics of human AGU disease.
U, Dunder +7 more
openaire +2 more sources
Enzyme replacement therapy for Anderson-Fabry disease
2010Anderson-Fabry disease is an X-linked defect of glycosphingolipid metabolism. Progressive renal insufficiency is a major source of morbidity, additional complications result from cardio- and cerebro-vascular involvement. Survival is reduced among affected males and symptomatic female carriers.To evaluate the effectiveness and safety of enzyme ...
Regina P, El Dib, Gregory M, Pastores
openaire +2 more sources
Renoprotection by enzyme replacement therapy
Current Opinion in Nephrology and Hypertension, 2004Barry M, Brenner, Jean-Pierre, Grünfeld
openaire +2 more sources
Enzyme replacement therapy for the mucopolysaccharide storage disorders
Expert Opinion on Investigational Drugs, 2002The mucopolysaccharide storage disorders are a group of lysosomal storage disorders associated with deficiencies of lysosomal enzymes required for the normal sequential degradation of glycosaminoglycans, formerly known as mucopolysaccharides. The accumulation of glycosaminoglycans in a wide variety of tissues results in a complex and progressive ...
openaire +2 more sources
Enzyme replacement therapy for Fabry's disease
The Lancet, 2010Stephen, Waldek +3 more
openaire +4 more sources
Prenatal Enzyme-Replacement Therapy
Obstetric Anesthesia Digest(N Engl J Med. 2022;387(23):2189–2193. doi: 10.1056/NEJMe2211515) Genetic diseases are difficult to treat, and though treatments have been developed none are curative. One still developing treatment is enzyme replacement therapy, and it is currently the standard of care for several lysosomal disorders. A recent study reported the first use of
openaire +1 more source
1983
Publisher Summary This chapter discusses the recent developments in the field of enzyme replacement therapy, describes the successes achieved, and indicates procedures that are expected to improve prospects for enzyme replacement therapy in the future.
openaire +1 more source
Publisher Summary This chapter discusses the recent developments in the field of enzyme replacement therapy, describes the successes achieved, and indicates procedures that are expected to improve prospects for enzyme replacement therapy in the future.
openaire +1 more source
Intrathecal enzyme replacement therapy.
2007A girl with Hurler's disease, diagnosed at the age of two years has been treated with weekly intravenous ERT since the age of 4 years. Although already having central nervous system involvement with psychomotor delay, she was still an active child with good communication skills.
De Meirleir, Linda +3 more
openaire +1 more source