COURTNEY BOGGS, A MEMBER OF THE CURE MITO FOUNDATION, WITH HUSBAND JACOB AND DAUGHTERS EMMA (LEFT) AND RILEY. EMMA, 6, WHO HAS LEIGH SYNDROME, CAN'T WALK WITHOUT ASSISTANCE AND EATS THROUGH A FEEDING TUBE. THE FOUNDATION HAS BEEN WORKING FOR A TREATMENT, BUT THE COMPANY IT PARTNERED WITH HAS PAUSED ITS WORK ON IT./RANDY BOGGS/KHN
Some families are forming their own biotech businesses to help their children, acting as drug developers to find treatments for ultra-rare diseases that affect 1,000 patients or fewer.