Will Genethon and Eukarÿs Partnership Reduce the Cost of Gene Therapies?
The FDA-approved only gene therapy for Duchenne muscular dystrophy is costing far too much for families to afford. Families simply cannot afford this prohibitive...
Belief BioMed Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate BBM-D101
Belief BioMed Reports FDA IND Clearance for Gene Therapy Candidate BBM-D101 for Duchenne Muscular Dystrophy.
The U.S. Food and Drug Administration (FDA) has approved an...
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...