Third Orphan Drug Designation Granted to TXA127 in Muscular Dystrophy

LAMA2-related muscular dystrophy accounts for 30–40% of cases of congenital muscular dystrophy
LAMA2-related muscular dystrophy accounts for 30–40% of cases of congenital muscular dystrophy

Tarix Orphan announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to TXA127 for the treatment of laminin-deficient congenital muscular dystrophy (LAMA2 MD or MDC1A).

Approximately 1 in 30,000 people develop early-onset LAMA2-related muscular dystrophy, which accounts for 30–40% of total cases of congenital muscular dystrophy.

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TXA127 previously received Orphan Drug designations for other muscular dystrophies including Duchenne's muscular dystrophy (DMD) and limb girdle muscular dystrophy (LGMD).

TXA127 is a pharmaceutical grade formulation of the naturally occurring peptide angiotensin (1-7).

For more information visit TarixOrphan.com.

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