Tragedy has struck during an important Pfizer medical trial, when a young boy suffered a fatal sudden cardiac arrest after receiving experimental gene therapy — a death that's sent scientists and parents scrambling to find out how this happened,DMD patients often have a mutated, dysfunctional version of the dystrophin gene, which makes the protein dystrophin — crucial for keeping muscles together in the human body.
The nonprofit patient advocacy group Parent Project Muscular Dystrophy reported that the patient received the gene therapy in early 2023, "We grieve this loss," he wrote. "There are no words to describe the unjust nature of a tragedy like this. Let us come together in this time of mourning, holding this Duchenne community near and dear as our hearts feel hollow and our minds search for answers."