Editor’s Choice: Duchenne, GSD and Orphan Drugs

Happy Friday!

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.

 

World Orphan Drug Congress: 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.

 

Rare Disease Classroom: Glycogen Storage Disease Type 1B

Glycogen storage disease type I is an inherited disorder caused by the buildup of a complex sugar called glycogen in the body’s cells.

 

Duchenne Muscular Dystrophy Therapy Enters Phase 3 Trial

FibroGen has just announced that their investigative therapy pamrevlumab has received Rare Pediatric Disease designation from the FDA.

 


Do you have a rare disease experience of your own? Share with us here.

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