By way of further encouragement, wanted to share below. Perhaps next year there is something in the list for our "disease".
https://blogs.fda.gov/fdavoice/inde...advances-and-looking-ahead-to-policy-in-2018/
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm
http://fortune.com/2018/01/02/new-drug-approvals/
https://blogs.fda.gov/fdavoice/inde...advances-and-looking-ahead-to-policy-in-2018/
https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm
http://fortune.com/2018/01/02/new-drug-approvals/