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This study found that enzyme replacement therapy in mice with a severe form of classical homocystinuria improved metabolic patterns and alleviated many clinical symptoms.

This narrative review highlights the critical role of intracrine hormone production in peripheral tissues, suggesting that continuous-release subcutaneous testosterone pellet therapy may offer a more effective symptom-driven approach for aging populations, though large-scale randomized controlled trials are needed for confirmation.

This review discusses recent advancements in therapies for ichthyosis, highlighting promising prospects in protein replacement and gene therapy, but it reports no new clinical results.

This review discusses the diverse clinical manifestations of mucopolysaccharidoses in children and emphasizes the importance of early diagnosis and treatment initiation, but it reports no new clinical findings.