Update of treatment for mucopolysaccharidosis type III (sanfilippo syndrome)
Weijing Kong, Yanqing Yao, Jing Zhang, Cheng Lu, Yingxue Ding, Yan Meng
Topics & Concepts
Enzyme replacement therapySubstrate reduction therapyMucopolysaccharidosisGenetic enhancementDiseaseLysosomal storage diseaseMedicineIncentiveOrphan drugBioinformaticsInternal medicineGeneBiologyGeneticsEconomicsMicroeconomicsLysosomal Storage Disorders ResearchBiomedical Research and PathophysiologyTrypanosoma species research and implications