Research and Innovation

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Mitochondrial disease therapy is still in its infancy. Mitochondria have been linked to human disease only since the 1960s, and much of what we know about these diseases has been discovered only in the past 20 years. Our knowledge about mitochondrial diseases is changing rapidly, as are the available tests and treatments.

In our Mitochondrial Program at Boston Children’s Hospital, we are experienced in evaluating children with mitochondrial disorders, anticipating the symptoms they will face and finding the best treatments for them. We take great care to stay ahead of the latest developments in the field and to thoughtfully consider all options in treating your child.

In all cases, we will work hand-in-hand with you and your family. We will listen carefully to your observations on whether treatments are working for your child and any side-effects he or she may be having, making adjustments as needed.

Research at Boston Children’s

Because so much needs to be learned about mitochondrial diseases, and because we want to provide better treatments for our patients, we are actively engaged in clinical research to develop and evaluate new treatments.

We offer an experimental medication, Dichloroacetate (DCA), to patients whose mitochondrial disease has caused lactic acidosis, a condition in which there is too much lactate in the blood and spinal fluid. This medicine has been proven to reduce the lactate levels and potentially alleviate disease symptoms. Boston Children’s is one of only a few institutions in the United States where this protocol is approved.

You can read more about the research going on throughout the Boston Children’s Neurology department here.

You can search for other clinical studies throughout Boston Children’s here.

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Contact the Mitochondrial Program

  • 617-355-2751
The future of pediatrics will be forged by thinking differently, breaking paradigms and joining together in a shared vision of tackling the toughest challenges before us.”
- Sandra L. Fenwick, President and CEO