Common-sense reform for the review of ultra-rare disease therapies by the Food and Drug Administration sounded like a dream come true for families like mine who are living with incurable, lethal childhood diseases. Today, I worry it could come too late to help my son.

My son, Declan, has Barth syndrome. This ultra-rare genetic mitochondrial disease affects fewer than 150 people in the United States. There are no approved treatments. An investigational drug that improves mitochondrial function, elamipretide, has shown promising results for some patients, including my 3-year-old son. This therapy improved his heart function, energy levels and overall health. Without it, we face an uncertain and frightening future.

In December 2023, I joined nearly 20,000 people to sign a petition urging the FDA to review elamipretide. In 2020, 2022 and 2024, more than 50 medical experts wrote to the FDA, urging it to act. Last October, the Cardiovascular and Renal Drugs Advisory Committee concluded that there is substantial evidence of effectiveness to support the approval of elamipretide for treating Barth syndrome.

This was tremendous progress for our small community after many years of educating different FDA review divisions about our ultra-rare disease and the urgent need for safe and effective treatment options.

I wholeheartedly support Health and Human Services Secretary Robert F. Kennedy Jr. and Dr. Marty Makary’s common-sense approach to making children suffering from chronic diseases healthy again — and quicker. I have been frustrated and heartsick at the delays that have long hindered our community’s access to this drug. Now, I worry my son will get caught in the crossfire of these essential reforms if critical decisions are delayed. Delays are not policy issues but a matter of health, safety and survival.

Every day without action is a day closer to losing the treatment that has given my son a future. Without FDA approval, critically ill children receiving this drug could lose access. Families could be forced to watch our boys’ health decline, not because science failed but because the FDA failed to act. I implore Kennedy and Makary not to let reform impede progress for my son and his “Barth brothers.”

I pray that Kennedy and Makary are poised, willing and able to step into the leadership breach during this critical period to ensure that drugs reviewed, debated and validated by experts do not fall through the bureaucratic cracks. I hope common-sense reforms do not come at the expense of my son and others like him.

Jamie Dubuque, the mother of a toddler with Barth syndrome, is a leader in the grassroots advocacy campaign Not Too Rare to Care/InsideSources