Chrissy was chosen as the first human being to ever receive this type of gene therapy delivered directly into the central nervous system. 












She underwent a week of outpatient workup in April with scans, tests, and samples of everything you could possibly think of. Amanda was an amazing little sister and more empathetic and loving than you've ever seen in a 5 year-old.


On May 27, 2015 Chrissy received the therapy. The injection was pretty anticlimactic and thankfully entirely uneventful. Unfortunately on her fourth day of hospitalization, Chrissy developed a rhinovirus infection. And what should have been a cold became pneumonia. Thankfully she was already in the ICU of the world's largest biomedical research institution.


She avoided intubation but required oxygen for what seemed like forever but was only about 10 days. She never let it get her down. A steady succession of visitors and family helped make sure her effervescent spirit never had a chance to even waiver. She became a wiz a ordering room service, of course with multiple desserts.


After almost 2 weeks in the hospital, Chrissy was able to join us back at home. Her homecoming was something we were looking forward to and couldn't wait for but when we were finally told she could go home we weren't expecting it but we didn't waste a single second. Amanda made a homemade "Welcome Home" sign for her sister of which she was deservedly proud.


Unfortunately Chrissy passed away in February 2016 due to complications of surgery, not related to the AAV9 virus injection.  (Please see Chrissy A Hero)


In October of 2016 Amanda was injected with the AAV9 virus.  In October of 2017 she returned to the NIH for her annual checkup and evaluation.


Much more research is needed to find a cure for this horrible disease.  The final cure has not been attained.