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Last Updated: 2024-05-21 (yyyy-mm-dd).

Title: Home Maggie s Pearl

Description: Based on the success of our foundational research with Perlara and Maggie s n of 1 treatment the FDA has approved our clinical trial for up to 40 PMM2 CDG children ages 2 17 We re partnering with the Mayo Clinic protocol includes telemedicine visits Research into treatments for other CDGs is planned

Language: English

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