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Pyruvate Dehydrogenase Complex Deficieny (PDCD) is a rare and life-limiting neurodegenerative disease. There are approximately 1,000 children suffering from PDCD in the U.S.
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There are currently 0 approved treatments for PDCD. If it had been approved, Sodium Dichloroacetate (DCA) would have provided significant quality of life improvements to children that desperately need it.
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The initial meeting with the FDA regarding DCA for the treatment of PDCD was over 12 years ago. Our kids can't wait any longer for access to this life changing medication.
Our families and kids are suffering.
PDCD does not wait.
Many children with PDCD die in infancy. Those that survive often face severe neurological, developmental, and physical challenges, including:
Severe Energy Depletion
Inability to Communicate
Intellectual
Disability
Developmental
Delay
Muscle Weakness / Limited Mobility
Irreversible Brain Damage
For more information on PDCD, check out the Hope for PDCD website
In September, the PDCD community was devastated by the news that Sodium Dichloroacetate (DCA) was not approved by the Food and Drug Administration (FDA). There were no safety concerns, the FDA essentially did not see the benefit.
PDCD is a rare disease, making it exceedingly difficult to produce any statistically significant information during a clinical trial.
For those that know these kids best, the moms, dads, caregivers, grandparents, and teachers; the quality of life benefits from DCA have been huge. We've seen increases in energy and alertness, less pain, and milestones reached that we never dreamed possible.
We are asking the FDA to please sit down with us and hear our lived experiences, to hear first hand what an approval of DCA would mean to our community. Our children's lives are at stake and they don't have time to wait.
DCA in the News
DCA Updates
PDCD Butter Challenge
11/22/2025
Spreading awareness about PDCD is crucial to helping us get DCA approved. You can help by participating in the 2025 Butter Challenge, beginning on November 22nd! More details coming soon.
Change.org Petition
10/30/2025
Join us in urging the FDA to approve DCA – the only treatment that has given PDCD kids stability and improved their lives – and to listen to PDCD families, meet our children, and hear our stories. This is not about lowering the bar for scientific evidence. It’s recognizing that for rare diseases, the bar must be different.
Saol Therapeutics Presentation
9/18/2025
Presentation on the DCA clinical trial, FDA decision, and what comes next. Huge thank you to both Saol Therapeutics and Hope for PDCD for putting on this extremely informative webinar.
Mito Advocacy groups respond to FDA's DCA denial
9/4/2025
United Mitochondrial Disease Foundation (UMDF), Mito Action, Cure Mito, Hope for PDCD, and the Elizabeth Watt Research Fund issue a joint statement and call to action.
Senate Hearing on Rare Disease Treatments
11/17/2025
The Senate Special Committee on Aging will be holding a hearing on rare disease treatment authorization. Mito advocacy groups sent a joint letter to the committee urging them to use the opportunity to elevate the voices of the PDCD community and the urgent need for DCA.
Physicians Letter to the FDA
10/8/2025
More than 80 of the leading researchers and clinicians in the mitochondrial disease space have signed a joint letter of support urging the FDA to pursue an expedited, flexible regulatory path forward for DCA
Saol Therapeutics News Release
9/8/2025
News release regarding the FDA's response to their DCA application.
Benefits of DCA
Below are just some of the benefits kids with PDCD have seen while taking DCA:
Increased Energy
PDCD severely affects energy, any increase in energy has a huge quality of life impact for these kids
Less Restrictive Diet
Most kids with PDCD are on a very strict ketogenic diet, less restrictions mean they can eat a healthier diet with more fruit and vegetables
Decreased Pain
Decreased pain means more good days. When time with your loved one is limited, the good days are priceless
Better Sleep
Poor sleep is very common with PDCD, some kids have slept through the night for the first time on DCA
Increased Alertness
Kids are better able to process and enjoy the world around them, accelerating their learning
Lower Lactic Acid
Lactic acid can build up in the brain and central nervous system, putting kids at risk for seizure, stroke, coma, or worse
Increased Life Expectancy
Typical life expectancy is early childhood, any extension of that is monumental to kids and their families
More Milestones Reached
Some kids have started walking, talking, and eating by mouth on DCA
