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Outcomes of Allogeneic Hematopoietic Cell Transplantation in Patients with Dyskeratosis Congenita
UploadHip Dysplasia in Patients With Hurler Syndrome (Mucopolysaccharidosis Type 1H)
Download from www.researchgate.netTALEN-based Gene Correction for Epidermolysis Bullosa
Download from doi.orgEnzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome
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