Anton SimeonovDrug discovery and development for rare genetic disordershttps://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.38326Gerald F. CoxTrials and Tribulations... Efficacy Endpoints for Clinical Trialhttps://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.38629Jonathan C. GoldsmithChallenges of Developing and Conducting Clinical Trialshttps://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.38413Hans D. OchsFrom Clinical Observations... Primary Immunodeficiency Disordershttps://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.38480James J. DowlingTreating pediatric neuromuscular disorders: the future is nowhttps://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.38418Albert R. La SpadaTherapy development in Huntington diseasehttps://onlinelibrary.wiley.com/doi/10.1002/ajmg.a.38494
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