The researchers have been able to get some really good results in the lab but the work is far from done. We need your help to cure this terrible disease and Hannah’s Hope Fund is the way to do it. Both of our children are really lucky they appear to have a slower progressing case of GAN due to their specific mutation. He is still able to walk.
The scientists working funded by Hannah’s Hope Fund have developed what they hope is a cure. It’s called a “GAN gene delivery” to the central nervous system and we hope a clinical trail gets approval to start as early as March of 2014. While the clinical trial will take place at the National Institutes of Health, there are many parts that Hannah’s Hope Fund will have to pay for. While the trial will get a healthy copy of the GAN gene to the central nervous system, Hannah’s Hope Fund is working to on a drug for the nervous system that may also be used to fight the effects of other diseases like ALS, Alzheimer’s Disease, Parkinson’s Disease, Frontal Lobe Dementia with Louis bodies, Alexander Disease and other forms of inherited neuropathy.
Justin is scheduled to be a part of the GAN Natural History Study at the National Institute of Health and will hopefully be chosen to participate in the clinical trial although he will not be in Phase 1 of the trial.