Successful matched unrelated donor hematopoietic stem cell transplantation for infantile Wolman disease

• Main Authors: Indira Jayakumar (Author), Anupama Gude (Author), Murarji Renangi (Author), Soundaram Valliyappan (Author), Venkateswaran Vellaichamy Swaminathan (Author), Satishkumar Meena (Author), Harika Varla (Author), Rumesh Chandar (Author), Ramya Uppuluri (Author), Revathi Raj (Author)
• Format: Book
• Published: Elsevier, 2023-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Introduction: Wolman disease is a rare genetic disorder with an autosomal recessive inheritance. A mutation in the LIPA gene causes lysosomal acid lipase (LAL) deficiency results in lipid storage and adrenal insufficiency. Death in early infancy is due to liver failure. Patients and methods: We desc...