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Innovation

A total of 432 posts are filed under Innovation
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
Rare Disease Report: RASRx is the Latest Duchenne Drug To Get Orphan Status From the FDA
Thanks to Rare Disease Report for this piece. University of Southern California (USC) start-up RASRx hopes to develop a drug to treat… 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
Video: How Children’s Hospital of Philadelphia is Helping Leukodystrophy Patients
Leukodystrophies are inherited disorders of the white matter of the brain and spinal cord that can cause problems with movement, speaking,… 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