Update on Pfizer’s DAYLIGHT Gene Therapy Trial for Duchenne

The Duchenne muscular dystrophy community recently faced a profound loss—a young participant in Pfizer’s Phase 2 DAYLIGHT study (for boys 2 years to less than 4 years of age) passed away due to cardiac arrest, over a year after receiving an investigational micro-dystrophin gene therapy aimed at treating Duchenne.

Decoding Gene Therapy for Duchenne

While there is currently no cure for Duchenne muscular dystrophy, gene therapy has the potential to target the underlying genetic cause of many mono genetic diseases and could potentially benefit […]