The Pediatric Motor Disorders Program at the University of Utah currently has 53 AHC families enrolled in our online registry! The AHC Registry allows us to quickly and efficiently contact families in the future with information about new treatments for AHC, or about studies for which they may be eligible. Additionally, this provides us a rough “head count” of all people with AHC. If you are interested in joining our online registry (and have not done so already), please visit our website: http://medicine.utah.edu/neurology/research/swoboda/ahc/Ongoing%20Research%20Studies.htm and click on “The AHC Registry.”
We also have 17 families who have completed our updated AHC medical questionnaire! The primary benefit of these questionnaires is knowledge gained regarding the characteristics of AHC in children and adults. There is no immediate personal benefit for participating in completing the AHC Questionnaire, however, we hope that information gained will help us in our search for effective therapies in AHC. The questionnaire can also be found on the above website and faxed back to our research office (fax: 801-587-9346). If possible, please complete the online registry before completing the questionnaire.
And last, but not least, our clinical trial of Sodium Oxybate in children with AHC is well underway and making progress. We currently have four participants enrolled and two in the screening process. For this pilot study, our total participation is capped at six. Three of the four participants have completed their six weeks on Sodium Oxybate and are in the follow-up stage of the study. The fourth participant is currently in the six-week initial drug phase. If our last two participants meet all inclusion and exclusion criteria we, hope to have them start the medication soon as well! This will provide us with the 6 participants we were aiming for.
We want to thank all the families who have participated in our studies. We greatly appreciate their time and dedication to AHC research. We also would like to invite all families who have not yet registered with our AHC Registry or filled out the updated medical questionnaire to please do so at http://medicine.utah.edu/neurology/research/swoboda/ahc/Ongoing%20Research%20Studies.htm