Giant Axonal Neuropathy (GAN) is a rare genetic disorder that slowly takes away one's ability to walk, use one's hands, speak, swallow and is terminal. We are a family driven 501C3 Public Charity, dedicated to funding research for a treatment and/or a cure for this horrible disorder. Our international team of scientists is working on a novel gene delivery approach that, if successful, can be applied to many closely related neurological genetic disorders like SMA and CMT, two other ravaging disorders that lack treatment options. Our scientific team is led by Dr. Steven Gray, in the Samulski lab at UNC at Chapel Hill's Gene Therapy Center. We are on target to begin a phase 1 clinical trial in 12-18 months. Please join our mission and help us save these precious lives.

 For scientific and other updates, please click the BLOG link at the top of this page.

Hannah's Hope Fund is funding a natural history study on GAN.  This study tracks the untreated course of the disease, with the goal of establishing clinical outcome measures that will be used in future clinical trials. Phase II clinical trials require that you establish how you will measure if an Investigational New Drug (IND) is slowing, accelerating or having no impact on disease progression.  Specifically, the outcomes measures obtained during the natural history study (pre-treatment) will be compared to post-treatment data sets obtained during clinical trials initiated to treat GAN.  To learn more, click here.

Please note that participation in this study is not for an investigational new drug, and participation in this study does not guarantee participation in future clinical trials.

"Never doubt that a small group of thoughtful, committed citizens can change the world.  Indeed, it is the only thing that ever has." - Margaret Mead