Had a good talk with Addysons motility specialist today. He said he wants to move her appointment with the aadp (autonomic dysfunction) clinic up. He said she’s urgent because of her cardiac issues. He also said he wants to really follow her gi /swelling stoma issues over the next 4 weeks. He thinks her stoma might need a revision. He needs to speak to her cardiologist about this Bp issue. If cardiology agrees he will move the NG to a G. This would be nice so she’s not throwing up NG tubes. He said he would not recommend another bowel resection which was offered. He said there is a major downfall to that plan and he just doesn’t feel it would be a good option for her long term. He also said if we can keep her stable and hang on there are two med options that should be available within the next year or two. He is hopeful one of them would work. The one is currently available now but only on a compassionate basis which he could get but it causes cardiac issues and he doesn’t think she could handle those risks right now.
He knows Addyson well. He talked with me for an hour about where we are, what his thoughts are and where he thinks we might be headed. These decisions are hard. There is no easy straight forward options.
He also talked to Addyson. She expressed her concerns and her Thoughts. He said he sees and hears the trauma she has about some of these invasive tests. He said the trauma is so real for These kids. He said it shows just how invasive all of this is. He heard her which is good. He acknowledged her fears.
Compassionate use means:
In certain situations, the Food and Drug Administration (FDA) allows companies to provide their experimental drugs to people outside of clinical trials. This is referred to as compassionate use. But getting access to not-yet-approved drugs through a compassionate use request can be a long and challenging process.
For you to receive an experimental drug through the compassionate use program, your doctor must contact the drug company and then submit an application to the FDA. For the FDA to consider your request, you must meet certain criteria:
• Your disease is serious or immediately life-threatening.
• No treatment is available or you haven’t been helped by approved treatments for your disease.
• You aren’t eligible for clinical trials of the experimental drug.
• Your doctor agrees that you have no other options and the experimental treatment may help you.
• Your doctor feels the benefit justifies the potential risks of the treatment.
• The company that makes the drug agrees to provide it to you.
Beaming with hope 