Hope you all are doing good.
My 20 month old son has been recently diagnosed with DMD(“A large deletion is seen in the dystrophin gene involving multiple exons(exons 2-44)”. Thought of posting here, in case anyone can let me know if any of the clinical trials that are currently going on can benefit my son?
I am in no means and in any position to give advice or medical expertise so take my comments with utmost reserve.
Due to your large deletion, especially in the first 10% of exons, exon skipping might not work for your son. There are plenty of other clinical and preclinical trials that might benefit your son in future, especially since he is so young.
There is always ISPC cells that come to mind in all cases, these are all preclinical trials and I am hoping that there are some updates this year. There are currently two labs working on this. One of them is Dr. Michele Calos, and the other is Dr. Rita Perlingeiro. Both of them seem to be extremely nice and are working diligently.
Second two options that come to mind are HT-100 from Halo Therapeutics, which is in Clinical Trials now, and SMT-C1100 that is supposed to go into Clinical Trials this year. Both of these are mutation independent and should benefit all DMD/BMD patients.
Really appreciate your response on this and thanks a lot for providing insights on other trials that are happening besides exon-skipping. this will really help as i can now followup these trials(IPSC, SMT100 and HT-100) as well.
Have a good day.