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PB1463 : ANALYSIS OF SURVIVAL IN PATIENTS WITH ACID SPHINGOMYELINASE DEFICIENCY TYPE B USING A LARGE, DEIDENTIFIED US ELECTRONIC HEALTH RECORD DATABASE

Researchers

Presenter

  • Amanda Wilson

Principal Investigators

  • Gandarvaka Miles

  • Krisha Patel

  • Mario Aguiar

  • Ruth Jacob

Medical Centers

  • Sanofi Genzyme,Reading,United Kingdom

  • Aetion Inc.,NY,United States

  • Sanofi Genzyme, Cambridge, MA, United States

Locations

  • United Kingdom

  • United States

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Endocrine/metabolic Diseases (ME)

  • Ophthalmology (OP)

Disease

  • Niemann-Pick disease type A

  • Anterior segment dysgenesis 1

Biomarkers

  • N/A

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • N/A

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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