On June 8th, Edison Pharmaceuticals announced that they have been given approval by the FDA to expand access to their orphan drug, EPI-743, which has already shown great promise in early clinical trials. You can read more about it in this article at CNBC.
I know of at least one child with Leigh’s Disease that has been involved in the clinical trial of this drug and it has improved her life tremendously! This could be huge and the first “real” treatment for mitochondrial diseases.
Everyone is now asking, of course, how do I get in the trial? How can I get this drug for my child? Answers to those questions will hopefully be coming next week, when Edison presents to audiences for MitoAction and the United Mitochondrial Disease Foundation.
On Thursday, June 16th, MitoAction will welcome Edison Pharmaceuticals to one of their teleconferences which is open to the public. Anyone can dial in and listen to learn more. (MitoAction also records all of their teleconferences and posts them on their website. We will link to that once it is posted after the call.) You can get the specific information about the call and how to participate, on MitoAction’s website.
On Friday, June 17th, the UMDF will welcome Dr. Bruce Cohen along with Edison Pharmaceuticals to an informational session at their Annual Mitochondrial Symposium (which is being held in Chicago this year). If you are going to the Symposium, you will be able to attend this informational session on Friday morning to learn more. (At the time of publication, it was not clear if UMDF will record the session and make it publicly available afterward.) You can get more specifics on the session on the UMDF website.