Up to now, the treatments works well, and his parents are trying to reduce the doses within the range that the doctor says fine. But it seems that he would never be able to live without medicine, esp. the hormonic drugs, which causes abesity and other discomforts. Thank you so much for being so considerate, it is true that some medical workers do not know how to communicate with patients properly. I feel so lucky to have a friend like you!
Is his autoimmune attack on his red blood cells aggressive or are they able to maintain his stability with treatments? We might be able to help you understand some of this if you run into some non-communitive doctors. Some of our medical friends seem to lack the ability to help others gain a clear understanding as to what's going on.
Thank you for telling me about the trial and your comforting words. Hope I could learn more good news from you. I would try my luck at the site that you offered. Thank you once again!
So terrible to be afflicted like this at the beginning of life. He's fortunate to have a caring family to do battle for him. I would call all the top medical centers and talk to their clinical trials group. Mayo Clinic, Cleveland Clinic, etc ... There must be something going on somewhere. Unfortunately rare diseases aren't generally spread out across the country due to the small number of availible participant pools, so travel might be inevitable.
Here's a web contact that I used myself to get in touch with several ongoing trials. Hopefully there's something you can find useful. Take care and we're all hoping that something wonderful happens! Many good research activities going on these days.
https://clinicaltrials.gov