Topic: Advances in Mucopolysaccharidosis and Alpha Mannosidosis

Mucopolysaccharidoses (MPS) and Alpha-Mannosidosis are rare lysosomal storage disorders characterized by the deficiency of specific lysosomal enzymes, leading to the accumulation of complex sugars. These disorders often present early in life with severe clinical symptoms that significantly impact patients' quality of life and lifespan. Despite recent advancements, diagnosing and treating these conditions still presents significant challenges.

The Special Issue "Advances in Mucopolysaccharidosis and Alpha-Mannosidosis" aims to compile and showcase the latest research findings and clinical practices to propel progress in diagnosing and treating these rare diseases. We invite researchers, clinicians, and industry experts to submit original research, reviews, and perspectives to share their latest discoveries and experiences in this field.
● Genetic and Molecular Insights: Discoveries related to gene mutations, enzyme deficiencies, and molecular pathways involved in MPS and Alpha-Mannosidosis;
● Diagnostic Innovations: Development of new biomarkers, advancements in genetic testing, and imaging techniques for early and accurate diagnosis;
● Therapeutic Approaches: Progress in enzyme replacement therapies, gene therapies, stem cell treatments, and new drug developments;
● Clinical Trials and Patient Care: Findings from clinical trials, patient management strategies, long-term follow-up studies, and assessment of treatment efficacy and safety;
● Emerging Technologies: Utilization of CRISPR gene editing, RNA interference, and nanotechnology in therapeutic interventions.

Through this Special Issue, we aim to foster interdisciplinary collaboration and inspire innovative thinking in the research and treatment of MPS and Alpha-Mannosidosis. We believe this issue will provide valuable insights for readers and drive advancements in this cutting-edge field.

31 Dec 2024