Cambridge-based Tarix Orphan LLC announced that U.S. FDA has granted Orphan Drug status to the company’s lead compound, TXA127, for the treatment of laminin-deficient congenital muscular dystrophy.  Early-onset LAMA2-related muscular dystrophy occurs in approximately 1 in 30,000 individuals and accounts for between 30 and 40 percent of total cases of congenital muscular dystrophy.   TXA127 is a pharmaceutical grade formulation of the naturally occurring peptide Angiotensin (1-­7), which Tarix Orphan is developing for the treatment of a number of orphan and genetic diseases, including congenital muscular dystrophies.  This is the third U.S. Orphan Drug designation granted to TXA127 for muscular dystrophies, with previously received designations for Duchenne’s muscular dystrophy (DMD), our lead indication, and limb girdle muscular dystrophy (LGMD).

 

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http://www.prnewswire.com/news-releases/tarix-orphan-granted-orphan-drug-status-for-txa127-as-potential-treatment-for-congenital-muscular-dystrophy-mdc1a-300220784.html