Eli’s mom, who had never heard of Hurler syndrome before the baby’s diagnosis, became distraught after reading about the condition. “It's literally like your heart falls out of your body ...

He was diagnosed with MPS1 Hurler Syndrome at the age of six months. Sam had a bone marrow transplant shortly after his first birthday. He's had more than 20 surgeries but has managed to stay ...

Hurler syndrome arises when a child inherits a flawed copy of a gene called IDUA from each ... including the brain, eyes, heart and bones. Enzyme replacement therapy can help with some of those ...

Since Hurler's syndrome involves organs known to be susceptible to RRT in HCT, including the brain, liver, heart and lungs, these patients might be expected to manifest a higher degree of ...

The Penn team also tested the gene editor on 10-week-old mice with Hurler syndrome, observing similar positive effects. Those mice did develop cardiac disease at four months of age, similar to ...

Hurler’s syndrome is a rare hereditary condition caused by the lack of an enzyme needed to regulate basic cell functions. ... and heart problems. Untreated children usually die by age 10.

BU Cp (P = 0.06) or marrow cell dose (P = 0.32) was not different in Hurler syndrome compared to other IMSD. ... cardiac or pulmonary dysfunction, or other potentially life-threatening problems.

LONDON--(BUSINESS WIRE)--Technavio has announced its pipeline analysis report on the Hurler syndrome market. The report includes a detailed analysis of the pipeline molecules under investigation ...

CINCINNATI (WKRC) - A family wants to raise awareness for their son’s rare genetic disorder. Titus Barrett-Weber has shown off his infectious smile since he was born 10 months ago. At such a ...