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Val-Rox Approval Favored in Europe, Stalled in US

BioMarin received a positive CHMP opinion after the FDA delayed val-rox's BLA filing for the second time.

Krystal Seeks Approval for Dystrophic Epidermolysis Bullosa Gene Therapy

B-VEC recently showed efficacy in the phase 3 GEM-3 trial.

Steven W. Pipe, MD, on Challenges in Gene Therapy Trials for Hemophilia

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed common challenges with gene therapy clinical trials.