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Science & Technology

A total of 155 posts are filed under Science & Technology
CheckRare: FDA Approves Haegarda to Treat HAE
The U.S. Food and Drug Administration today approved Haegarda, the first C1 Esterase Inhibitor (Human) for subcutaneous administration to… Continue Reading
Patient-Inspired Research Uncovers New Link to Rare Disorder
Meeting a young patient with Zellweger syndrome, a rare, life-threatening genetic disease, started a scientific investigation that… Continue Reading
The Atlantic: What If (Almost) Every Gene Affects (Almost) Everything?
By Ed Yong (SOURCE) In 1999, a group of scientists scoured the genomes of around 150 pairs of siblings in an attempt to find genes that are… Continue Reading
Dalhousie Researcher Studying Drug to Combat Fabry Disease
A simple pill that switches off a genetic trigger could potentially provide a powerful treatment for Fabry disease, a rare disorder that… Continue Reading
Genentech to Highlight Actemra and Rituxan Data at EULAR Annual Congress
Various new data on Genentech’s Actemra (tocilizumab) and Rituxan (rituximab) will be presented at the 2017 European League Against… Continue Reading
Castle Creek Announces First Patient Enrolled in DELIVERS Study of Diacerein 1% Ointment for Epidermolysis Bullosa Simplex
Castle Creek Pharmaceuticals (CCP), a global company dedicated to delivering transformative therapies to patients with orphan dermatologic… Continue Reading
Amicus Therapeutics Receives Rare Pediatric Disease Designation for SD-101 for Patients with Epidermolysis Bullosa
 Amicus Therapeutics, Inc.(link is external) (Nasdaq:FOLD), has received Rare Pediatric Disease designation from the U.S. Food and Drug… Continue Reading