FDA Grants Rare Pediatric Disease Designation to TXA127
Tarix Orphan’s orphan drug TXA127 received a Rare Pediatric Disease Designation from the FDA for the treatment of recessive dystrophic epidermolysis bullosa.

TXA127, is a formulation of the natural Angiotensin (1-7) peptide which is designed to interfere with the TGF beta pathway.

It is also being tested as a treatment for Duchenne muscular dystrophy (DMD). A Phase 2 study in DMD patients is expected to begin in early 2017.

At this time, it is not known when a clinical trial for epidermolysis bullosa will begin....

http://www.raredr.com/news/fda-grants-rare-pediatric-disease-designation-to-txa127
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