"To fund great leaps in research, there needs to be an urge to succeed, " said Tolar.

CNN: A child's struggle inspires drive for cure

In January, Martin Tolar, a former Pfizer executive, joined Knome as its new CEO.

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"It is a misconception that rare disease research only helps a small number of patients, " said Tolar.

CNN: A child's struggle inspires drive for cure

Still, Tolar admits that his greatest allies in finding support for his research are his patients and their families.

CNN: A child's struggle inspires drive for cure

"The fact is, we need more brainpower to develop new therapies, " said Tolar.

CNN: A child's struggle inspires drive for cure

"I'm not saying this is a cure, " said co-author Dr. Jakub Tolar, an associate professor of pediatrics at the University of Minnesota.

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Since then, Tolar has transplanted a total of 24 children with EB, and continues to conduct research in gene therapy for EB and other rare diseases.

CNN: A child's struggle inspires drive for cure

Tolar applauds this type of fundraising.

CNN: A child's struggle inspires drive for cure