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Niagen Bioscience Receives Exclusive U.S. FDA Rare Pediatric Disease (RPD) Designation and European Medicines Agency Orphan Medicinal Product Designation (OMPD) for NB4168 for the Treatment of Ataxia Telangiectasia (A-T)

Contributed by: Business Wire

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FDA
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Children
General Health
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Ataxia Telangiectasia

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The parable of the lost sheep mirrors our journey to find a solution for Duchenne Muscular Dystrophy (DMD), a rare disease. Our global interdisciplinary team is fortunate to have been gifted with Neu REFIX, whose hidden potentials of beneficial immune modulation, we unravelled by extensive preclinical and clinical studies. This gives hope to DMD patients for its potentials in improving their quality of life by halting the disease progress. Like the shepherd when he found the lost sheep, our joy knew no bounds, when we got encouraging outcome in DMD research. Now on receiving orphan drug and rare paediatric disease designations from US FDA, while it’s time to rejoice, we have miles to go until it reaches every deserving patient after an approval, the final goal. We are open to collaborate with likeminded institutes to reach the goal. Further insights have revealed the potentials of Neu REFIX in Psoriasis, auto-immune diseases and in Multiple Sclerosis by gut microbiome reconstitution. (Graphic: Business Wire)

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