Rare Leader: Vanessa Vogel-Farley Executive Director Dup15q Alliance

The Basics Name:  Vanessa Vogel-Farley Title:…

Fighting a Cold War
In 2009, the U.S. Navy deployed Jodie Christopher to Djibouti, Africa as an electronic technician to program radios and work on radar… Continue Reading
Aeglea BioTherapeutics Presents Positive Interim Clinical Data in Ongoing Phase 1/2 Trial of Pegzilarginase in Patients with Arginase 1 Deficiency
Rare Daily Staff In mid-October, Aeglea BioTherapeutics, a company that designs and develops innovative human enzyme therapeutics for… Continue Reading
Abeona Therapeutics Licenses RegenxBio’s AAV9 Vector to Develop Gene Therapies for Four Rare Lysosomal Storage Disorders
Rare Daily Staff Abeona Therapeutics, a company focused on developing novel gene and cell therapies for life-threatening rare diseases,… Continue Reading
Novo Nordisk Acquires U.S. and Canadian Rights to Strongbridge Biopharma’s Oral Diagnostic for AGHD
Rare Daily Staff Strongbridge Biopharma, a company focused on the development and commercialization of therapies for rare diseases, has… Continue Reading
Researchers Unravel a Genetic Network Linked to Autism
Rare Daily Staff University of Toronto Donnelly Centre researchers have uncovered a genetic network linked to autism. The findings, which… Continue Reading
Inaugural Familial Chylomicronemia Syndrome Awareness Day—November 2, 2018
Rare Daily Staff Akcea Therapeutics announced that November 2, 2018 marks the inaugural Familial Chylomicronemia Syndrome (FCS) Awareness… Continue Reading
GW Pharma’s Cannabidiol Therapy Now Available for Rare Childhood Onset Epilepsy
Rare Daily Staff GW Pharmaceuticals’ recently approved medicine Epidiolex is now available in the United States for the treatment of… Continue Reading

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