A couple articles on parents starting drug companies to try to help their children. One Dad’s 14-Year Quest for Cure http://www.wsj.com/articles/one-dads-14-year-quest-for-childrens-cure-1419639957 This article is about Pompe/Amicus/John Crowley For those interested I would recommend the book by the same author its a little bit old (also movie available) not too heavy on the science as I recall and almost reads like a drama/novel. When Parents Start Companies to Cure Their Children http://www.wsj.com/articles/when-parents-start-companies-to-cure-their-children-1419639500 Don't envy anyone needing to be in that position but Wall St. Journal has a few links of organizations that may help get you started at least. http://blogs.wsj.com/briefly/2014/12/26/when-your-childs-rare-disease-has-no-cure-the-short-answer/