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!
