• Allogeneic Hematopoietic Stem C
  • 1 In the past 25 years, nearly a thousand patients with these types of storage disorders, including mucopolysaccharidosis (MPS) type I (Hurler syndrome), other MPS, adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD), Krabbe disease, and others have received allogeneic hematopoietic stem cell transplantation (HSCT) with bone marrow from matched or mismatched related donors who were either carriers or noncarriers of the disease, resulting in clinical benefit in many of them. (bloodjournal.org)
  • donor
  • When the healthy stem cells from an unrelated donor, that do not exactly match the patient's blood, are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. (clinicaltrials.gov)
  • 2 ⇓ ⇓ ⇓ ⇓ ⇓ ⇓ ⇓ ⇓ ⇓ ⇓ ⇓ ⇓ ⇓ - 16 The benefit is primarily derived from the replacement of missing enzyme produced by donor cells circulating in the blood and also from engraftment of donor-derived glial cells in the brain. (bloodjournal.org)