FDA Grants Rare Pediatric Disease Designation to Nippon Shinyaku’s Exon 51 Skipping Study (NS-051/NCNP-04)
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...
Elevidys Gene Therapy has Arrived in Brazil and Could Cost up to 20 Million Brazilian Reais
There is no definitive treatment for Duchenne muscular dystrophy (DMD). The Elevidys gene therapy is a treatment method that slows down the disease, and...
3 Drugs Receiving Accelerated Approval from the FDA and Reviewed by the Office of Inspector General Raised Concerns
Despite shaky evidence and reviewer differences, the US Food and Drug Administration swiftly approved Aduhelm from Biogen, Exondys 51 (eteplirsen) from Sarepta, and Makena...
Sarepta Reiterated Total Net Product Revenue of $3.0 Billion in 2025
As children and families battling Duchenne Muscular Dystrophy wait for drug prices to drop, Sarepta has announced its earnings forecast for 2025. How much...
Wave Life Sciences Announces Plans for Exon 53 Skipping Study in 2025
Alpha-1 antitrypsin deficiency (AATD), Huntington's disease (HD), obesity, and Duchenne muscular dystrophy (DMD) are among the major unmet medical needs that the company seeks...