"Like so many rare disease organizations, Maggie's PMM2-CDG Cure is family-driven and a force of nature," said Ethan Perlstein, Ph.D., CEO of Perlara. "We will develop the first invertebrate (yeast, nematode, fly) drug-screening pipeline for PMM2-CDG in three phases of research with the goal of discovering lead compounds with pharmaceutical properties suitable for cost-effective safety and efficacy studies in the recently described Pmm2R137H/F115L mouse model."
"We couldn't be more excited to be working with a true innovator in Perlara," said Holly and Dan Carmichael, Founders of Maggie's PMM2-CDG Cure. "Their dedication and devotion to work with families to find cures was evident on our first call. Our daughter, Maggie, has an amazing spirit and is working hard – be it in physical therapy or in her school – on a daily basis. As parents we feel we owe it to her to do whatever we can to find a treatment and we are overjoyed to be working with Perlara on Maggie's journey."
Earlier this month, Perlara announced its first two PerlQuest partnerships. There are currently a total of six disease programs at the company. Next month, Perlara relocates from its home of the last three years in the biotech incubator QB3@953 to a newly constructed, 13,000+ sq. ft. lab and office space in South San Francisco, the birthplace of biotechnology.
Perlara is a scientific discovery public benefit corporation that works with families and drug companies around the globe to find treatments for diseases previously believed to be too rare to cure. For general information and updates, please visit us at perlara.com, connect with us at @PerlaraPBC on Twitter and at www.facebook.com/PerlaraPBC on Facebook, and learn specifically about PerlQuests at www.perlara.com/perlquests and www.perlara.com/faqs.
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