6.29.2017
Meeting a young patient with Zellweger syndrome, a rare, life-threatening genetic disease, started a scientific investigation that… Continue Reading
6.25.2017
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
6.23.2017
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
6.22.2017
A simple pill that switches off a genetic trigger could potentially provide a powerful treatment for Fabry disease, a rare disorder that… Continue Reading
6.14.2017
Various new data on Genentech’s Actemra (tocilizumab) and Rituxan (rituximab) will be presented at the 2017 European League Against… Continue Reading
6.14.2017
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
6.11.2017
Castle Creek Pharmaceuticals (CCP), a global company dedicated to delivering transformative therapies to patients with orphan dermatologic… Continue Reading
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