It’s not easy. The word Duchenne enters our lives. We search the internet, seek out expertise, connect with researchers, clinicians, industry, other families, set up our internet tools (Google alerts, RSS feeds, list-serves, pubmed search criteria, industry alerts, etc) and live each day hoping to see our ‘in-box’ overflowing with news, with HOPE.
On any given day, you reassure yourself that your son is young, may have a certain mutation, may have a certain degree of muscle function, may have what it takes to participate in a promising clinical trial. You connect with experts in the field as an insurance policy for today and tomorrow, to protect and preserve strength. You connect with others who are in the ‘know,’ who may recommend supplements, approved drugs that they believe have some degree of effect. This may be based on a study in the mdx mouse, a clinical trial for another condition and/or a guess, based on knowledge of the downstream pathology of Duchenne. It may be a response to the question “if this was your son, what would you do?” posed to a physician, researcher or a parent with experience or knowledge and/or coerce your doctor to write a script for a drug on the basis of mdx data and use in other populations. How many times have we all said "It won’t hurt and may help, so let’s try.”
On another day, you may find yourself dragging around because, for some reason, it hits you that everything takes time, too much time and you wonder, “Will it be in time for my son?” Or you walk around the entire day praying that your son walks or that your son continues to move his arms, continues to breathe without ventilation, continues to breathe, continues to have a healthy heart.
On another day, your son falls, or it’s the day of the school IEP, or he is no longer walking or someone says the wrong thing or you read about a trial design that involves only ambulatory boys and you find it hard to talk, hard to hold back tears. Or your son has a fever and your heart stops in its tracks, worried about cold, flu, pneumonia.
On another day, a random person in the media will use words like 'breakthrough' or ‘cure’ or ‘promising treatment’ and you wonder…Is it real?
Hope is a good thing, but it is often impossible to find the right balance, to take in the overwhelming amount of information and make sense of it… make sense of it for your life and what it means for your son.
As a community, we have a great deal of experience with dose escalation. HOPE - Trials, each of us working hard to find our own therapeutic dose.
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