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FDA approves Phase III trial 
for exciting new PMM2-CDG treatment 

 

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Phase III epalrestat trial begins soon…

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Families

 

Want to learn more about the PMM2-CDG clinical trial or our ongoing CDG research?

Partners

 

Would you like to become one of our research or clinical collaborators?

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Based on the success of our foundational research with Perlara PBC and compassionate use trial of one patient in the US, the FDA has approved a prospective, single-center, randomized, double-blind, placebo-controlled clinical trial at Mayo Clinic for up to 40 PMM2-CDG children (ages 2-17).

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Your clinical trial experience

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The study will be conducted at the Mayo Clinic by  experienced medical and research staff.

 
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The science

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Decades of safety data from epalrestat's use in Japan and India to treat nerve damage in adults with diabetes has supported the application for studying it’s use in children with PMM2-CDG.

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Word
on the street

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The results of our subject in the "pioneer" study and the approval to start the Phase III trial have attracted the attention of the scientific and business press. Join in the conversation.

"The improvement of PMM enzyme activity, and global glycosylation suggest epalrestat is a rational treatment target for PMM2-CDG and should be tested in a larger clinical trial."

Sorbitol is a severity biomarker for PMM2-CDG with therapeutic implications

Annals of Neurology

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Collaborative science

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Founded upon science and collaboration between researchers, clinicians and families, Maggie’s Pearl is starting a clinical trial for PMM2-CDG patients to determine if epalrestat is safe and effective.

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Latest News

Groundbreaking biotech gets closer to possible treatment for kids with a rare disease

Word on the street

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It's official

Check out the official government
clinical trial page

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$2M Grant

Maggie's Pearl wins

"Direct-to-Phase 2" SBIR award

Image of Mayo Clinic Rochester Minnesota, Gonda Building and part of Mayo Building from cafeteria Copyright ©2008 Michael Bonert.

You are free to share and adapt this image as per the CC BY-SA 3.0.