Patients with Hurler's syndrome
showed an increase in blood leukocyte Idua enzyme, the enzyme that is deficient in untreated patients with Hurler's syndrome
, suggesting that transplant with MGTA-456 is beginning to affect the disease in these patients.
A 6-year-old girl with known Hurler's syndrome
presented to our outpatient department with Umbilical Hernia since two and a half years.
"There are two current treatments for Hurler's syndrome
: costly enzyme replacement therapy or bone marrow transplants which require a perfectly matched donor," said Dr Sharon Byers, affiliate senior lecturer within the School of Molecular and Biomedical Sciences.
is the severe form of MPS type1, or mucopolysaccharidoses.
For many of the inborn errors of metabolism, BMT functions as permanent enzyme replacement therapy In Hurler's syndrome
, the most commonly studied, up to 80% of patients are cured if BMT is done prior to onset of organ dysfunction.
Citation: "Outcomes of transplantation using various hematopoietic cell sources in children with Hurler's syndrome
after myeloablative conditioning"; J.
is a lysosomal storage disorder that affects approximately one of every 100,000 children.
is a rare hereditary condition caused by the lack of an enzyme needed to regulate basic cell functions.
Robyn - the face of the campaign - suffers from the terminal illness Hurler's syndrome
and her parents regularly use Scotland's only children's hospice, Rachel House, near Kinross.
causes mental and physical disability.
Lindsey Devine, whose daughter Katie has a rare genetic condition called Hurler's syndrome
, was invited to attend the Royal College of Nursing to contribute to the project.
(ATHX) said a published scientific article describes positive results of a preclinical study in mice evaluating the administration of its stem cell product for the treatment of MPS-t (or Hurler's Syndrome