Search Results

Twice weekly dosing with Sebelipase alfa (Kanuma®) rescues severely ill infants with Wolman disease.

• Authors : de Castro, María José; Jones, Simon A; de las Heras, Javier

Subjects: INFANT diseases; OXYSTEROLS; SURVIVAL rate

• Source: Orphanet Journal of Rare Diseases; 6/25/2024, Vol. 19 Issue 1, p1-9, 9p

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Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease.

• Authors : Potter, Jane E.; Petts, Gemma; Ghosh, Arunabha

Subjects: HEMATOPOIETIC stem cells; STEM cell transplantation; STEM cell treatment

• Source: Orphanet Journal of Rare Diseases; 5/21/2021, Vol. 16 Issue 1, p1-14, 14p

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Clinical insights from Wolman disease: Evaluating infantile hepatosplenomegaly.

• Authors : Hannah, William B.; Ryan, Katherine; Pendyal, Surekha

• Source: American Journal of Medical Genetics. Part A; Nov2022, Vol. 188 Issue 11, p3364-3368, 5p

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A 14‐step desensitization protocol for sebelipase alfa hypersensitivity in a patient with Wolman disease and secondary hemophagocytic lymphohistiocytosis.

• Authors : Nguyen, Minh H. N.; Bruening, Rachel; Abel, Trent

Subjects: MEDICAL protocols; HEMOPHAGOCYTIC lymphohistiocytosis; GLYCOGEN storage disease type II

• Source: Pediatric Allergy & Immunology; Apr2024, Vol. 35 Issue 4, p1-4, 4p

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Wolman disease presenting with hemophagocytic lymphohistiocytosis syndrome and a novel LIPA gene variant: a case report and review of the literature.

• Authors : Asna Ashari, Kosar; Azari-Yam, Aileen; Shahrooei, Mohammad

Subjects: LITERATURE reviews; HEMOPHAGOCYTIC lymphohistiocytosis; GENETIC variation

• Source: Journal of Medical Case Reports; 8/29/2023, Vol. 17 Issue 1, p1-9, 9p

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A New Mutation in Diagnosis of Wolman Disease: Case Report.

• Authors : Cansever, Mehmet Şerif; Aslan, Mine; Zübarioğlu, Tanyel

Subjects: GENETIC mutation; WOLMAN disease; CHOLESTEROL esters

• Source: Journal of Academic Research in Medicine; 2019, Vol. 9 Issue 4, p150-152, 3p

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First LIPA Mutational Analysis in Egyptian Patients Reveals One Novel Variant: Wolman Disease.

• Authors : Elaraby, Nesma M.; Galal, Eman Reda; Abdel-Hamid, Mohamed

• Source: Journal of Molecular Neuroscience; Aug2023, Vol. 73 Issue 7/8, p598-607, 10p

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Santiago de Compostela University Researchers Update Current Data on Wolman Disease [Twice weekly dosing with Sebelipase alfa (Kanuma(R)) rescues severely ill infants with Wolman disease].

Subjects: INFANT diseases; RESEARCH personnel; LYSOSOMAL storage diseases

• Source: Clinical Trials Week; 7/15/2024, p1621-1621, 1p

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Rapid whole‐genome sequencing leading to specific treatment for two infants with haemophagocytic lymphohistiocytosis due to Wolman disease.

• Authors : Selvanathan, Arthavan; Forwood, C.; Russell, J.

• Source: Pediatric Blood & Cancer; Sep2023, Vol. 70 Issue 9, p1-4, 4p

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Children's Medical Center Reports Findings in Wolman Disease (Wolman disease presenting with hemophagocytic lymphohistiocytosis syndrome and a novel LIPA gene variant: a case report and review of the literature).

Subjects: LITERATURE reviews; HEMOPHAGOCYTIC lymphohistiocytosis; GENETIC variationTEHRAN (Iran)

• Source: Pediatrics Week; 9/16/2023, p129-129, 1p

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