MPS II Archives - MPS Society

DHHS adds MPS II to the RUSP for Newborn Screening in the United States

August 3, 2022 - The National MPS Society announces that Xavier Becerra, the Secretary of the Department of Health and Human Services (DHHS) has approved adding MPS II as a condition to the recommended uniform screening panel (RUSP) for newborns. This long-awaited acceptance follows the Advisory Committee on Heritable Disorders in Newborns and Children’s (ACHDNC) approval to move the nomination forward to DHHS after a considerable evidence review.

Shire’s MPS II clinical trial fails to meet endpoints

Company will review data; analyze positive trends beyond initial results Shire announced today that top-line data from its phase II/III clinical trial for MPS II did not meet primary or … Continued

Sangamo treats first patient with gene therapy

Sangamo Therapeutics treated its first patient in the Phase 1/2 clinical trial (“the CHAMPIONS study“) evaluating SB-913, an investigational in vivo genome editing therapy for people with MPS II, Hunter … Continued

Induced-neuronal (iN) cells as tools to study the pathogenesis of neurological manifestations in MPS-II

Mucopolysaccharidosis type II (MPS-II) is a genetic disease caused by the inability to breakdown large molecules called glycosaminoglycans. MPS-II is caused by the deficiency of an enzyme located in the … Continued

Development of pharmacological chaperone therapy for MPS II.

We aim to develop novel drugs for MPS II with the potential to reach and effectively treat the brain. Our approach is via so-called Pharmacological Chaperone Therapy (or PCT), also … Continued

Adjunctive therapy for Hurler syndrome

Enzyme replacement therapy (ERT) remains an essential way to treat MPS disease. This therapy works because lysosomal enzymes made in the lab can be taken into cells and delivered to … Continued

AAV mediated gene transfer to the CNS for MPS II

Currently there is no established treatment for neurologic symptoms of Hunter syndrome, caused absence of the enzyme iduronate sulfatase (IDS). We have established conditions for effective gene transfer to all … Continued