for her parents, college professors patrick carr and maria cafalas, coming to terms with the disease was wrenching. and that's difficult. >> i remember the social worker said it's good to try to cry in the shower to save it from your family and your children. >> pull. pull. >> reporter: after more than a year of rage and grief maria decided that she had to find some way of helping mld children. it was too late for cal. but she was desperate to give her daughter's life meaning. >> we're not we didn't know very influential people who could write a big check for a million dollars. so we said we'll start selling cupcakes. >> reporter: the caliope joy foundation was formed in 2013. >> they're so good. >> reporter: it's been slow going. but the money added up. and when maria learned about the italian trial and the fact that amy price had to keep going back to milan with giovani and ceci, money to help families get to italy. >> she sent me a picture of giovani playing in his front yard. he's three months younger than cal. so he should have been as cal. he should be on a feeding tube. he sho

. >> reporter: maria cafalas has turned cupcakes into weapons of war. her war against mld. >> selling the company seems kind of silly, but i don't know what else to do. >> reporter: she's raised more than $250,000 and helped where she could. but she's hit a wall. so far not a single gene replacement therapy has been approved by the fda. the trial in italy is closed to new patients. it could be years before any children with mld will be allowed to receive the treatment in the united states. now it's like when do we get this here? what will it take? tell me what you need me to do. >> reporter: until then she continues to fight her battles -- >> yay! >> reporter: -- one cupcake at a time. the price children proof to her the war can be won. >> you keep using the word "miracle." in what way is all of this a miracle? >> our son is still with us. that's the miracle. hings in your home for up to 48 hours? it's like having a sick family member that you didn't even know was there. and we all know what happens when one family member gets sick. but lysol spray and

i need to help this happen again and again and again. >> reporter: maria cafalas has turned cupcakes into weapons of war. her war against ml kind of silly, but i don't know what else to do. >> reporter: she's raised more than $250,000 and helped where she could. but she's hit a wall. so far not a single gene replacement therapy has been approved by the fda. the trial in italy is closed to new patients. it could be years before any children with mld will be allowed to receive the treatment >> now it's just impatience. now it's like when do we get this here? what will it take? tell me what you need me to do. >> reporter: untheil tn she continues to fight her battles -- >> yay! >> reporter: -- one cupcake at a time. the price children proof to her the war can be won. >> you keep using the word "miracle." in what way is all of this a miracle? >> our son is still with us. that's the miracle. ceci fight heartburn fast. with tums chewy delights. the mouthwatering soft chew that goes to work in seconds to conquer heartburn fast. tum tum tum tum. chewy delights. only from tums. (achoo!) did y