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

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.