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. We are a completely volunteer organization with NO paid employees. Please join our mission and help us save these precious lives. Fantastic News for Children with GAN World-Wide!
Ms. Doris Buffett's Sunshine Lady Foundationprovided a matching grant for all funds raised between August, 2009 and February, 2010, up to $500,000!
Click the second box on the top right to see "Help Cure GAN." This video was made by one of our GAN children's Uncles.
Hannah's Hope for Giant Axonal Neuropathy (GAN) While 5 yr. old Hannah Sames is the inspiration for Hannah’sHope Fund, we very quickly realized a therapy to stop this ravaging disease is replicable and applies to any genetic disorder that impacts the central nervous system (brain and spine). Fortunately, scientists have made great gains with virus vectors that transport healthy genes to nerve cells, replacing the damaged or mutated gene. In Giant Axonal Neuropathy, it’s the GAN gene that is replaced with gene therapy. Hannah’s Hope Fund has raised enough funding to provide seed funding for a proof-of-concept study for GAN gene therapy. However, the following phases of this gene therapy project need significant external funding:
Studies to determine the most effective AAV serotype and route of administration of the viral vector
Studies to accomplish the necessary FDA efficacy and safety studies in order to gain approval for a Phase 1 clinical trial.
Financial support for the clinical trial
Once our clinical trial is proven successful, the same virus vector can be used to transport any gene to treat other genetically based neurological disorders like, Spinal Muscular Atrophy, Charcott Marie Tooth Disease, Freidrich’s Ataxia and Infantile Neuroaxonal Dystrophy, to name a few. We are so very anxious to move forward quickly to satisfy this unmet medical need. However we need funding in order to execute the plan we've devised with the best scientists in the world collaborating on this project. Thousands will benefit for generations to come.
"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
