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!
Our bi-annual conferences give families affected by MPS & related diseases an opportunity to learn about new research, treatment and care, and to share information and experiences with other affected individuals and families. The event was enjoyed virtually on...
As part of the Virtual Education and Connection Series, listen to our very special speakers talk about their unique and resilient stories regarding a rare disease...
