MavriX Bio announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) to its investigational gene therapy, MVX-220. Rare Pediatric Disease Designation is granted by the FDA to therapies intended to treat serious or life-threatening diseases that primarily affect individuals from birth through 18 years of age and affect fewer than 200,000 people in the United States.
This designation recognizes both the significant unmet medical need for individuals living with Angelman syndrome and the potential of MVX-220 as a one-time, gene replacement approach to treatment.
Read the press release: HERE
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