This week we have details about an investigative therapy for Duchenne Muscular Dystrophy that has entered a phase 3 clinical trial. Also, have you ever heard of Glycogen Storage Disease Type 1B? If not, we’ve prepared a crash course on this very rare disease. And, finally, if you missed the World Orphan Drug Congress on April 28, we’ve got the lowdown on what was discussed in the world of rare disease trials and patient registries.
On April 28th, the virtual World Orphan Drug Congress USA 2021 featured a variety of subjects relevant to the development of orphan drugs and the rare disease community.
Glycogen storage disease type I is an inherited disorder caused by the buildup of a complex sugar called glycogen in the body’s cells.
FibroGen has just announced that their investigative therapy pamrevlumab has received Rare Pediatric Disease designation from the FDA.