FDA Grants Rare Pediatric Disease Designation to Nippon Shinyaku’s Exon 51 Skipping Study (NS-051/NCNP-04)

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Nippon Shinyaku declared that NS-051/NCNP-04, a medication being developed to treat Duchenne Muscular Dystrophy (DMD), has been granted Rare Pediatric Disease Designation by the Food and Drug Administration (FDA).

Nippon Shinyaku and the National Center of Neurology and Psychiatry jointly discovered the antisense oligonucleotide NS-051/NCNP-04. By omitting a portion of the dystrophin gene’s genetic material, NS-051/NCNP-04 creates a functioning dystrophin protein with a little shorter chain length, which should prevent the decline in muscle function.

With a strong sense of purpose, Nippon Shinyaku has been actively developing medications to cure uncommon and intractable diseases in order to quickly introduce solutions for DMD patients.

Read More: Clinical Trials for Duchenne (List of All Researches)

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