Rare Leader: David Faughn, Director, NUBPL Foundation

The Basics Name:  David Faughn Title: Director,…

Retrophin Names ViiV Executive as Its New President and CEO
Rare Daily Staff Rare disease drug developer Retrophin said it appointed Eric Dube as president and CEO, effective January 4, replacing… Continue Reading
Cydan Launches Tiburio Therapeutics to Develop Treatments for Rare Neuroendocrine Tumors and Endocrine Diseases
Rare Daily Staff Orphan drug accelerator Cydan said it has launched Tiburio Therapeutics to focus on developing treatments for rare… Continue Reading
Rare Leader: Kelly Maynard, President and Founder, Little Hercules Foundation
The Basics Name: Kelly Maynard Title: President and founder Organization: Little Hercules Foundation Disease focus: Duchenne muscular… Continue Reading
Allena Pharmaceuticals Says It Has Finalized Design with FDA for Pivotal Trial in Hyperoxaluria Using Novel Endpoint
Rare Daily Staff Allena Pharmaceuticals said that in consultation with the U.S. Food and Drug Administration it has finalized the design of… Continue Reading
What, Me Worry?
The U.S. Food and Drug Administration’s Center for Drug Evaluation and Research set a record in 2018 by approving 86 orphan drugs,… Continue Reading
U.S. Food and Drug Administration Orphan Drug Designations in December 2018
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FDA Orphan Drug Approvals in 2018
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