A new study shows a 30-minute, inexpensive, and easy-to-use test may help measure the activity of NDST1, one of the main enzymes involved in heparan sulfate, the sugar molecule that build ups and causes Sanfilippo syndrome. Sanfilippo syndrome, or MPS III, is a...
We are disappointed to hear that Abeona Therapeutics has decided to end enrollment for their clinical trial (Study ABT-003) for MPS IIIA children aged 2-18 years with a Developmental Quotient score less than 60. The company has recently conducted a review of...
Courageous Parents Network is a non-profit organization and educational platform that orients, empowers and accompanies families and providers caring for children with serious illness. They have videos, podcasts, printable guides, Guided Pathways, and a blog to help...
MPS I (Hurler syndrome) Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations of Mucopolysaccharidosis Type I Hurler Syndrome (MPS-IH). Read the full report > SiglionTherapeutics, Development of a...
Read Gianna’s story and how it changed the course of her life. Newborn screening gave Gianna a head start in her fight against MPS I. Today marks the 1-year anniversary of Gianna’s bone marrow transplant. So amazing!
