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Dr. Cristin Davidson and Dr. Steven Walkley

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NPC Researchers

Dr. Cristin Davidson has been researching treatments for NPC for a decade, including cyclodextrin, a treatment she discovered in early 2006. Dr. Steven Walkley has spent more than 30 years researching lysosomal diseases like NPC.

Biography

Cristin Davidson is a post-doctoral fellow at the Albert Einstein College of Medicine. Steven Walkley is the director of the Rose F. Kennedy Intellectual and Developmental Disability Research Center and of the Dominick P. Purpura Department of Neuroscience at Albert Einstein College of Medicine in New York City.

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Cyclodextrin

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Discovery of Cyclodextrin

"The solubilizer was having the effect, not the allopregnanolone...it was a serendipitous discovery."

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Results of Cyclodextrin

"It is by far the most dramatic of any drug that's ever been used in this disease."

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Researching Cyclodextrin

Collaboration

Drs. Davidson and Walkley coordinate with SOAR (Support Of Accelerated Research), a collaboration of multiple labs dedicated to developing NPC treatments.

Ototoxicity

One of Drs. Davidson and Walkley's main focuses is developing a cyclodextrin treatment that doesn't cause hearing loss.

Further Investigation

Drs. Davidson and Walkley have been working hard to refine their understanding of cyclodextrin in order to make it even more effective for NPC treatment.

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FDA Approval: Vtesse Trial

"It's really critical to do this trial, to do it properly...so that [cyclodextrin] would be widely available for NPC patients."