In previous studies of bone marrow transplantation in patients with Hurler's syndrome, the median age was older — 19 to 24 months. 9,11,13,14 Children who undergo transplantation at a younger ...

Patient characteristics. From January 1986 to December 2001, we performed 30 consecutive transplants on 27 patients with Hurler's syndrome: 15 girls and 12 boys aged 11 months (m) (median) at the ...

One of those patients is 20-year-old Sam Caswell, who lives with his parents and dog Charlie in Bedford, NH. He was diagnosed with MPS1 Hurler Syndrome at the age of six months.

A deficiency of β-galactosidase (pH 5.0) was found in frozen tissues (brain, liver, kidney and spleen) from 10 patients with Hurler's syndrome (Types 1–3). The diminished activity of this ...

Hurler syndrome has no cure, but treatment that was first tried in the 1980s can prolong a patient’s life. Gupta knows of a person in her 30s living with the disease.

Hurler syndrome—a severe form of the lysosomal storage disease mucopolysaccharidosis type I (MPS I)—is caused by a deficiency or absence of the enzyme iduronidase, causing complex sugars to ...

Short stature is characteristic of Hurler syndrome, or mucopolysaccharidosis type IH (MPS IH). Hematopoietic stem cell transplantation (HSCT) is used to treat children with MPS IH. While HSCT ...

Orchard Therapeutics, a Kyowa Kirin company, today announced the last patient has been treated in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic ...

Gene therapy being developed as a one-time treatment to address the underlying cause of the disease by inserting a functional copy of the human IDUA gene into a patient’s own hematopoietic stem ...

Patients with the disease, called Hurler syndrome, suffer from short stature, spinal defects, and extremely stiff joints, complications that greatly limit their quality of life. advertisement ...

CALABASAS, Calif., Nov. 30, 2017 /PRNewswire/ -- ArmaGen, Inc., a privately held biotechnology company focused on developing groundbreaking therapies to treat severe neurological disorders, today ...

Hurler Syndrome can attack a patient's organs, joints, eyes and more. It was first recognized by German pediatrician Gertrud Hurler in 1919; doctors are still studying the condition.