GRAND RAPIDS, Mich. — For families of children with pyruvate dehydrogenase complex deficiency (PDCD), a rare metabolic disorder, hope is tied to one drug: DCA. The treatment is given orally and works to help the body process lactic acid, a key factor in the disease.
Right now, the FDA hasn’t approved the drug because the statistics in the trial were not proven to be beneficial enough, despite it passing all required safety protocols. “The families want the FDA to consider the human impact, not just the numbers in trials.”
Holly Cieslinski and Layna O’ Connor, mothers to Lillian & Piper, both children with PDCD, explained the urgency: “For these families, approval of the drug isn’t just medicine — it’s hope for more birthdays, more milestones, more moments together.”…