RGX-202 gene therapy meets main goal in Phase 3 DMD clinical trial
WHY IT MATTERS
If RGX-202 is approved, it could offer DMD patients—who currently have limited treatment options—a potential one-time gene therapy that addresses the root genetic cause of their muscle weakness.
A new gene therapy called RGX-202 has successfully completed its main testing goal in a Phase 3 clinical trial for Duchenne muscular dystrophy (DMD), a serious muscle-weakening disease. Gene therapy works by delivering healthy copies of genes into the body to fix genetic problems. This positive result suggests the therapy could become an important treatment option for DMD patients.
The Phase 3 portion of a clinical trial testing RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD), met its main goal, the therapy’s developer, Regenxbio, announced in a press release. Steve Pakola, MD, Regenxbio’s chief medical officer, said the data “support the potential of RGX-202 to become a best-in-class gene therapy for Duchenne […] The post RGX-202 gene therapy meets main goal in Phase 3 DMD clinical trial appeared first on Muscular Dys
ASK YOUR DOCTOR
Ask your neurologist or DMD specialist if you or your child might be eligible for RGX-202 trials or if this therapy could be appropriate once approved, especially if you have a confirmed dystrophin gene mutation.