ANVISA Cancels Elevidys Registration in Brazil
Brazil’s National Health Surveillance Agency (ANVISA) has cancelled the registration of Elevidys, a gene therapy used to treat Duchenne muscular dystrophy (DMD), following an...
FDA Allows Expanded Enrollment in Signature Biologics’ DMD Cell Therapy Study
Signature Biologics has announced that the U.S. Food and Drug Administration (FDA) has allowed expanded enrollment in a clinical study evaluating the company’s investigational...
IPS HEART Aims to Restore Full-Length Dystrophin with Two Regenerative Cell Therapies for Duchenne Muscular Dystrophy
IPS HEART has announced another important regulatory milestone after receiving FDA Rare Pediatric Disease Designation for ISX9-CPC, its investigational cardiac muscle therapy for Duchenne...
GEN6050X Exon 50 Skipping Base Editing Drug: One-Year Results Show Encouraging Progress for Duchenne Muscular Dystrophy
The GEN6050X exon 50 skipping base editing drug, developed by GenAssist Therapeutics, is attracting growing attention as one of the first base editing therapies...
Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen, an Exon 51 Skipping Therapy for Duchenne Muscular Dystrophy (DMD)
Families affected by Duchenne muscular dystrophy (DMD) received encouraging news as Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted...