Clinical Trial Update

//Clinical Trial Update

Clinical Trial Update

hannahNote from BLOG Admin: Hannah has been in Chapel Hill this week with her parents for pre-clinical trial testing:

Yesterday, Hannah had anesthesia for an MRI, NCV and LP to take CSF (cerebral spinal fluid). Her back is a little sore. She has no restrictions and will tells us if her back hurts. She also had tons of evaluations (PT, OT, cognative, hearing, BAER).

We had a great meeting today with our scientists and the doctor overseeing the clinical aspects of our clinical trial and Natural Disease History Study on GAN, which began this week. Hannah and 14 other kids will participate in the study over the next 24 months. The study charts the untreated course of the disease. The FDA requires this data as proof that the treatment is effective and safe for long-term use. The cost of this 2 year study is over $470,000.

Each of the participating children will have 5 visits for the same testing and evaluations Hannah underwent this week. Treatment can begin before all 5 visits occur. The pre-treatment data sets are compared to post-treatment data to assess efficacy and safety of treatment. The timeframe for beginning our clinical trial will depend on the FDA. Scientifically, the next 10 weeks are very busy, as critical efficacy studies will be wrapping up.

We are having monthly conference calls with our scientists and will have a scientific meeting in NYC at the beginning of March.

All of this is possible because of the countless hours of support, your generosity and love. THANK YOU.

With gratitude,
Lori & Matt

By |December 9th, 2010|Blog|Comments Off on Clinical Trial Update