This discussion is to hopefully stay connected with other families of boys who participated on the Phase 2a (38 boys in three sites), and to communicate with all the DMD community the progress of our boys while on PTC124.
My son Daniel Castro (10 years old) participated in the 28 days clinical trial of PTC124 along with other 37 boys a couple of years ago. Since, we have been patiently wating for the extension study. Thank God as of last week, we got the confirmation from Dr. Finkel's team in Philadelphia that everything is ready to go, and they are asking ALL the families of the boys who participated to request the weeks in which they would prefer to go to start the new study. If you are one of those families and you haven't gotten in contact with your hospital in which you did the original trial, please contact them ASAP to get this going for our boys. Great news is that our boys will be taken PTC124 for almost 2 years. They will need to get muscle byopsies again, but that is an small price to pay for the potential benefit we all expect to get out of it.
I encourage parents and families in general of these boys to use this discussion to talk about anything related to the development of this new study, and to report progress, complications, experiences, syntoms, differences noticed while in treatment etc. I think it would be good not only for those 38 families, but hopefully for ALL the kids with non-sence mutation who are participating on the new 2b study and even for all other DMD kids with other type of mutations since PTC therapeutics is working on a new medicine for ALL the cases of DMD.
God bless you ALL.
Jose, Angie, Daniel and the Castro Gang.