Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy Dyne Therapeutics, today announced that the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD). Today, Dyne Therapeutics, a clinical-stage company dedicated to developing life-changing treatments for individuals with genetically driven neuromuscular diseases, announced that DYNE-251 has been designated as an orphan drug by the European Commission (EC) for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being tested in DMD patients who are susceptible to exon 51 skipping in the Phase 1/2 DELIVER global clinical study.

Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy

#dmd #duchenne #dyne #dyne-251 #exon51 #exon51skipping #exonskipping #ema #orphandrug

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

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

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#dmd #duchenne #bmd #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #exon51 #exon51skipping #nipponshinyaku #exonskipping