Doing it All - How Families are Reshaping Rare Disease Research

Comparing and Validating Machine Learning Models for Mycobacterium tuberculosis Drug Discovery

(2018) Thomas Lane et al.   MOLECULAR PHARMACEUTICS

Industrializing rare disease therapy discovery and development

(2017) Sean Ekins   NATURE BIOTECHNOLOGY

Open Source Bayesian Models. 2. Mining a “Big Dataset” To Create and Validate Models with ChEMBL

(2015) Alex M. Clark et al.   Journal of Chemical Information and Modeling

A meta-analysis of randomized double-blind clinical trials in CMT1A to assess the change from baseline in CMTNS and ONLS scales after one year of treatment

(2015) Jonas Mandel et al.   Orphanet Journal of Rare Diseases

Genome Editing-Enabled HTS Assays Expand Drug Target Pathways for Charcot–Marie–Tooth Disease

(2014) James Inglese et al.   ACS Chemical Biology

Identification of Drug Modulators Targeting Gene-Dosage Disease CMT1A

(2012) Sung-Wook Jang et al.   ACS Chemical Biology