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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
DCA is filling a critical unmet need
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 Updates
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
