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Hannah's Hope Fund continues mission of eradicating GAN

But, Hannah's Hope Fund is not done yet, Favaro said.

"We're not where we need to be yet, so we can't really take time to celebrate what we've done," she said. "We've still got a ways to go."

Through events downstate where Hannah and her family live to ones locally like the annual Hannah's Hope Fund Benefit Dinner at the Rainbow Wedding and Banquet Hall, every penny counts, said Hannah's mother.

Currently, Hannah's Hope Fund is funding a natural history study on GAN that charts the untreated course of the disease, with the goal of obtaining clinical outcome measures.

"This is required by the [Food and Drug Administration] in order to begin a clinical trial," said Sames, who also serves as executive director of Hannah's Hope Fund. "The outcome measures obtained from the natural history study are used to measure efficacy of treatment during the clinical trial."

The FDA requires a plan of how to measure if an experimental drug or drug product is reversing, slowing, stopping or having no impact on the progression of a disease, said Sames. The research of GAN so far has yielded promising results, she added.

"On the research front, our scientists received far better than expected results for the number of nerve cells in the spinal cord we will be able to treat with a healthy copy of the GAN gene," she said. "Now, studies are under way to make certain the gene that gets delivered will express enough of the missing protein to be therapeutic."

Over-expression can be fatal and under-expression will have "little impact," added Sames, making this "the most critical piece of gene therapy" with "no margin for error."

The FDA requires Hannah's Hope Fund begins its research with the first phase being a safety trial consisting of consenting adults. A second phase would begin four months later in order for children to receive gene therapy.

Though GAN gene therapy isn't expected to have an effect on subjects who are completely paralyzed, it is expected to restore function to the nerves that are not yet dead.

"Therefore, every day that goes by without treatment is detrimental," Sames said. "We are trying to move fast."

For more information about Hannah's Hope Fund and how you can help, visit www.hannahshopefund.org.

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