Neurocognitive outcomes of intrathecal enzyme replacement therapy and transplant in Hurler syndrome

2018 ◽  
Vol 123 (2) ◽  
pp. S41
Author(s):  
Julie B. Eisengart ◽  
Weston Miller ◽  
Alex Kaizer ◽  
Kyle Rudser ◽  
Kelly King ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Hurler Syndrome ◽  
Enzyme Replacement ◽  
Neurocognitive Outcomes

scholarly journals Long-term cognitive and somatic outcomes of enzyme replacement therapy in untransplanted Hurler syndrome

2017 ◽  
Vol 13 ◽  
pp. 64-68 ◽  
Author(s):  
Julie B. Eisengart ◽  
Jeanine Jarnes ◽  
Alia Ahmed ◽  
Igor Nestrasil ◽  
Richard Ziegler ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Hurler Syndrome ◽  
Enzyme Replacement

scholarly journals Enzyme Replacement Therapy and Hematopoietic Stem Cell Transplantation Results in Patients with Hurler Syndrome: Clinical Cases

2019 ◽  
Vol 18 (3) ◽  
pp. 196-202
Author(s):  
Nato D. Vashakmadze ◽  
Leyla S. Namazova-Baranova ◽  
Natalia V. Zhurkova ◽  
Ekaterina Yu. Zakharova ◽  
Svetlana V. Mikhaylova ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Cell Transplantation ◽  
Hurler Syndrome ◽  
Hematopoietic Stem ◽  
Enzyme Replacement ◽  
Mps I

Mucopolysaccharidosis type I (MPS I) is the hereditary disease characterized with alpha-L-iduronidase activity decrease and further accumulation of heparan and dermatan sulfate in lysosomes. MPS I is rare autosomal recessive disorder with incidence of 0.5–4 cases on 100.000 live-birth infants. Meantime there two approaches in MPS I treatment: hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT). HSCT can be the best option for treatment of patients with severe MPS I (Hurler syndrome). Successful engraftment moderates such clinical signs as obstructive airway diseases, hepatosplenomegaly, cardiovascular system dysfunctions. HSCT prevents cognitive functions decline and other pathologic features of central nervous system. Presented clinical cases show various clinical courses according to age of diagnosis, ERT onset and HSCT implementation.

Enzyme replacement therapy with α-L-iduronidase and lectin RTB fusion protein in treating murine Hurler syndrome

2016 ◽  
Vol 117 (2) ◽  
pp. S88 ◽  
Author(s):  
Li Ou ◽  
Walter Acosta ◽  
Brenda L. Koniar ◽  
Renee D. Cooksley ◽  
Carole L. Cramer ◽  
...  
Keyword(s):  
Fusion Protein ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Hurler Syndrome ◽  
Enzyme Replacement

Does extending enzyme replacement therapy after transplant provide neurocognitive benefit in Hurler syndrome?

2021 ◽  
Vol 132 (2) ◽  
pp. S43
Author(s):  
Blake Gimbel ◽  
Ashish Gupta ◽  
Paul J. Orchard ◽  
Troy Lund ◽  
Julie B. Eisengart
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Hurler Syndrome ◽  
Enzyme Replacement

Clinical outcomes of Hurler syndrome treated exclusively with enzyme replacement therapy from a young age

2015 ◽  
Vol 114 (2) ◽  
pp. S40 ◽  
Author(s):  
Julie B. Eisengart ◽  
Elsa G. Shapiro ◽  
Kyle D. Rudser ◽  
Alia Ahmed ◽  
Victor Kovac ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Clinical Outcomes ◽  
Young Age ◽  
Hurler Syndrome ◽  
Enzyme Replacement

scholarly journals High-dose enzyme replacement therapy in murine Hurler syndrome

2014 ◽  
Vol 111 (2) ◽  
pp. 116-122 ◽  
Author(s):  
Li Ou ◽  
Tyler Herzog ◽  
Brenda L. Koniar ◽  
Roland Gunther ◽  
Chester B. Whitley
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
High Dose ◽  
Hurler Syndrome ◽  
Enzyme Replacement
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