New treatments for the mucopolysaccharidoses: from pathophysiology to therapy

• المؤلفون الرئيسيون: Simona Fecarotta (مؤلف), Serena Gasperini (مؤلف), Giancarlo Parenti (مؤلف)
• التنسيق: كتاب
• منشور في: BMC, 2018-11-01T00:00:00Z.
• الموضوعات: article
• الوصول للمادة أونلاين: Connect to this object online.

Abstract Enzyme replacement therapy is currently considered the standard of care for the treatment of mucopolysaccharidoses (MPS) type I, II, VI, and IV. This approach has shown substantial efficacy mainly on somatic symptoms of the patients, but no benefit was found for other clinical manifestation...