Samarco Raymond YI Background Lysosomal storage Disorders are a group of orphan Diseases Orphan diseases are diseases that affect less than 1 in 200000 people Some of the diseases are more prevalent in certain populations and Bias towards a gender ID: 931455
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Slide1
Alexion Challenge
Jayson Chen, Michael
Samarco
, Raymond YI
Slide2Background
Lysosomal storage Disorders are a group of orphan Diseases
Orphan diseases are diseases that affect less than 1 in 200,000 people
Some of the diseases are more prevalent in certain populations and Bias towards a gender
Slide3Challenges
Rarity of diseases makes it difficult to recruit patients for clinical trials
What is the likely distribution of the Disease?
What family should be prioritized?
Slide4Tools
Python – generate synthetic data.
Excel – compile information on diseases and make charts
BASH – command line processing
Slide5Approach
Due to rarity and nature of the diseases, data on affected peoples was sparse
Compiled data(Disease, rarity, bias) in excel
From estimates of prevalence, we generated synthetic data based on prevalence of disease into a text file using python
Slide6Results
Slide7Results