gargoylism

(redirected from Hurler syndrome)
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Related to Hurler syndrome: Hunter syndrome

gargoylism

[′gär·gȯi‚liz·əm]
(medicine)
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In contrast, in our study, samples from patients with Hurler syndrome (n = 7) show markedly increased concentrations of both DS and HS compared with controls (Fig.
Receipt of rare pediatric disease designation strengthens ArmaGen's commitment to providing children, families and physicians with treatment options for the currently unaddressed complications of Hurler syndrome," said James Callaway, Ph.
Dosing the first patient in the AGT-181 study represents another important step toward our goal of providing patients and physicians with treatment options for the currently unaddressed complications of Hurler syndrome," said James Callaway, Ph.
Bone marrow transplantation for globoid leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy and Hurler syndrome.
Stem cells derived from umbilical cord blood are currently a treatment option for over 40 diseases, including cancers such as acute lymphoblastic leukemia and Non-Hodgkins lymphoma, certain bone marrow failure syndromes such as severe aplastic anemia and neuroblastoma, certain blood disorders such as sickle cell anemia and other metabolic diseases such as Hurler syndrome.
ZFN-Mediated In Vivo Genome Editing Results in Supraphysiological Levels of Lysosomal Enzymes Deficient in Hunter and Hurler Syndrome and Gaucher Disease -- Abstract #479 Session: Gene Editing and Gene Regulation II Oral Presentation -- Friday, May 15, 2015
In recent years, Kurtzberg has expanded cord blood's application to treat a wide range of genetic diseases, like Krabbe Disease and Hurler Syndrome, which cause progressive physical and cognitive deficits leading to death in early childhood.
a privately held biotechnology company focused on developing novel therapies to treat severe neurological disorders, announced today that the Investigational New Drug Application (IND) for the company's second product candidate, AGT-181 for the treatment of Hurler syndrome, has been accepted by the U.
MPS I encompasses three related disorders, the most severe form of which is Hurler syndrome, a crippling and fatal disease that affects young children.
The best known and most severe form of MPS I, Hurler syndrome, is a crippling and fatal disease that affects young children.
The most severe form of MPS I, called Hurler Syndrome, is a crippling and fatal disease that affects young children.
Department of Biomedical Engineering at Yale University, will attempt to correct the gene disorder in Hurler syndrome in mice by "editing" genes in utero using nanotechnology.