Latest Advances

  • Condition: An Infant with Pelizaeus-Merzbacher Disease (PMD)
  • Journal: Medicine
  • Treatment Used: Rehabilitative Management
  • Number of Patients: 1
  • Published —
This case report discusses a one-month boy with Pelizaeus-Merzbacher disease (PMD), an X-linked recessive trait and a rare disease characterized by abnormal myelin formation in the central nervous system, treated with rehabilitative management.
  • Condition: Pediatric Patients with Inherited Leukodystrophies (LD)
  • Journal: Blood advances
  • Treatment Used: Cord Blood Transplantation
  • Number of Patients: 169
  • Published —
This study defined risk factors and describe the early and late outcomes of patients with globoid cell leukodystrophy, X-linked adrenoleukodystrophy, and metachromatic leukodystrophy undergoing cord blood transplantation (CBT).
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