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Thoo Neuromuscular Disease Neuromuscular Disease Drug: RGX-202 Company: RegenxBio Indication: Duchenne muscular dystrophy (DMD) Stage: Phase III Background: RGX-202 uses an AAV8 vector delivering a novel micro-dystrophin gene, including the C-terminal domain, which offers a potential improved dystrophin stability and muscle membrane integrity in DMD patients

In other words, it blocks the pathway for excess melanin