Spotting Birds of a Feather

John Atkinson became a birder in fifth grader when…

Perlara Enters Collaboration with the University of Notre Dame Focused on Cori Disease and Pompe Disease
Rare Daily Staff Perlara said it has entered into a collaboration with the University of Notre Dame and the Warren Family Research Center… Continue Reading
Sigilon Therapeutics Names Rare Disease Veteran Rogerio Vivaldi as CEO
Rare Daily Staff Sigilon Therapeutics said that it has named Rogerio Vivaldi as its CEO to replace Paul Wotton, who has stepped back from… Continue Reading
Orchard Therapeutics Raises $150 Million to Advance Gene Therapy Pipeline
Rare Daily Staff Orchard Therapeutics said it completed an oversubscribed series C financing of $150 million to advance its gene therapy… Continue Reading
FDA Approves Amicus’ Fabry Therapy Galafold
Rare Daily Staff The U.S. Food and Drug Administration today approved Galafold, the first oral medication for the treatment of adults with… Continue Reading
FDA Approves Alynlam’s RNAi Therapeutic for hATTR Amyloidosis
Rare Daily Staff The U.S. Food and Drug Administration approved Alnylam Pharmaceuticals’ Onpattro, a first-of-its-kind RNA interference… Continue Reading
Rare Leader: Michele Erwin, President and Founder of All Wheels Up
The Basics Name: Michele Erwin Title: President and founder Organization:  All Wheels Up Focus:  We are focused on the funding and… Continue Reading
FDA Grants Fast Track Designation to Castle Creek Pharmaceuticals EBS Treatment
Rare Daily Staff The U.S. Food & Drug Administration has granted Fast Track designation to Castle Creek Pharmaceuticals’ CCP-020 for its… Continue Reading
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