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'We're grateful we took that chance': 8-year-old with rare, fatal disease shows dramatic improvement on experimental treatment
By Nicoletta Lanese
Child walks again after receiving experimental treatment for rare genetic condition
Coenzyme Q10 Precursors Reverse Motor Decline in a Young Patient
By Stephanie DeMarco
Experimental Compound Offers Potential Treatment for Rare, Often Fatal, Childhood Disease
Full Coverage of this story