A Rare Disease Advocate Teaches Doctors to See and Speak Differently

When Rachel Callander’s daughter Evie was born…

FDA Expands Approval of Vertex’s CF Drug Kalydeco to Include Kids 12 to Less than 24 Months
Rare Daily Staff The U.S. Food and Drug Administration expanded the approval of Vertex Pharmaceuticals’ cystic fibrosis drug Kalydeco to… Continue Reading
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
Mustang Bio Enters License Agreement with St. Jude for X-SCID Gene Therapy
Rare Daily Staff Mustang Bio said it entered into an exclusive worldwide license agreement with St. Jude Children’s Research Hospital for… 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

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