The positive results obtained in Hurler syndrome
and few other LSDs and the benefit observed in our on going Phase I/II clinical trial of HSC gene therapy for the demyelinating LSD metachromatic leukodystrophy indicate that migration of the transplanted Hematopoietic Stem Cells (HSCs)/their progeny into the affected human brain occurs.
com), is studying the use of laronidase (Aldurazyme) for Hurler syndrome
, Scheie syndrome, and Hurler-Scheie syndrome.
He added the unit in Crumlin could also be used for less rare diseases "such as Hurler syndrome
, primary immunodeficiencies and sickle cell disease".
Eight-year-old son Wyatt Blancheri has Hurler syndrome
, a rare genetic disorder that progressively damages his organs and brain.
MPS I is one of the most common lysosomal storage diseases to affect the central nervous system, which in severe form can become Hurler syndrome
Mum-of-five Michelle Rodden, 39, said she had been forced to leave her 19-year-old ADHD suffering son in her house, while moving to the council-run building with 16-year-old pregnant daughter Stacey, 14-year-old Stephanie, Leighton, nine, and seven-year-old Demi-Leigh, who suffers from Hurler syndrome
2-5) A search of Medline from 1966 to the present with the search words Hunter, mucopolysaccharidosis, thickening, cervical, and/or cord did not yield reports of cervical cord thickening in patients with Hurler syndrome
(OMIM 607014) (1) and Hunter disease (OMIM 309900) (2, 3), also referred to as mucopolysaccharidoses types I and II (MPSIH and MPS II),  are caused by deficiency of the enzymes [alpha]-L-iduronidase (EC 3.
Cardiovascular manifestations of the Hurler syndrome
The lysosomes in the cells of children with Hurler syndrome
do not have a vital enzyme called IDUA (/a-/L-idunronidase), which causes their cells to accumulate too much of a class of biochemical known as mucopolysaccharides, in this instance dermatan sulfate and heparin sulfate.
He was diagnosed with MPS 1 - Hurler Syndrome
at a few months old and doctors said it was terminal without a bone marrow transplant.
In January 2003, Jayne and Anthony were told Thomas had a life-threatening condition called Hurler syndrome