428 top medical experts on Lysosomal Storage Diseases across 48 countries and 37 U.S. states, including 248 MDs (Physicians). This is based on an objective analysis of their Scientific Publications, Clinical Trials, Medicare, and NIH Grants.

  1. Lysosomal Storage Diseases: Inborn errors of metabolism characterized by defects in specific lysosomal hydrolases and resulting in intracellular accumulation of unmetabolized substrates.
  2. Clinical guidelines are the recommended starting point to understand initial steps and current protocols in any disease or procedure:
  3. Broader Categories (#Experts): Inborn Errors Metabolism (1,547) and Narrower Categories: Aspartylglucosaminuria (58), Cholesterol Ester Storage Disease (235), Cystinosis (1,047), Mannosidase Deficiency Diseases (22), Mucopolysaccharidoses (1,530), Nervous System Lysosomal Storage Diseases (88), Pycnodysostosis (330).
  4. Clinical Trials ClinicalTrials.gov : at least 35 including 4 Active, 10 Completed, 8 Recruiting
  5. Synonyms: Lysosomal Enzyme Disorders


  

        

                    


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