Mucopolysaccharidoses and similar lysosomal disorders - A guide for affected individuals, their families, doctors and nursing professionals

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Focusing on lysosomal storage disorders, particularly mucopolysaccharidoses, this updated guide addresses recent advancements in diagnostics and therapies for these rare diseases. It covers the pathophysiology and offers a comprehensive overview of treatment options like bone marrow transplantation and enzyme replacement therapy. Aimed at health professionals, affected individuals, and families, the book serves as both a reference and a supportive resource for understanding and managing these complex conditions.