Tomorrow morning we set out on a journey across the country to start a new drug trial they call the Biomarker Validation for Niemann-Pick Disease, type C: Safety and Efficacy of N-Acetyl Cysteine at the National Institute of Health in Bethesda, MD. This clinical trial will hopefully lay the groundwork for future therapeutic trials. You can read more about it here (I’ll spare you from me trying to explain it): http://www.nnpdf.org/NPCClinicalTrialOpportunity.html.
Brisan and Parker also started on November 23rd weekly infusions of DDVAP which has been used for completely different reasons but they have found that it helps elevate your Von Willibrand Factor which contributes to helping your blood clot. As some of you may know they both have an issue with clotting with a underlying bleeding disorder that they have not been able to pinpoint exactly. The weekly infusions will hopefully allow them to clot faster therefore keeping the sanity in the house!
As parents it is frustrating not only because your child doesn’t understand why you have to hold their nose “forever” but it always happens at the wrong times! Stay tuned about more info on the NAC trial and updates!
Dad
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