Welcome to the
PMM2-CDG Phase III Trial

We’re making fast progress and invite you to join us in breaking through to a new treatment.

We’re setting out to change the path for drug development and have shown that in under 5 years you can go from nothing to a phase III drug trial of a potentially significant therapy, the first ever for PMM2-CDG. A toast to the end of 2021 was due after the FDA gave the official “STUDY MAY PROCEED” approval for a 40 patient trial taking place at the Mayo Clinic sponsored by Maggie’s Pearl.




Modeling and drug candidate discovery

Rapid model development and high throughput screening finds a drug candidate


IND study

FDA approved N-of-1 epalrestat trial shows dramatic improvement in small group of patients

Phase III
clinical trial

FDA approves epalrestat Phase III trial for 40 patients




What to expect


Personalized clinical trial experience

Our trial partners, including Mayo Clinic, have a long history providing a supportive, premium participation experience. Maggie’s Pearl is with you throughout the trial process, and stays with you afterwards with updates and services.

Your comfort
our priority

We appreciate your participation in the trial. You are our guest. Hotel and travel accommodations will be reimbursed. We want your participation to be as seamless and low in stress as possible.


to make a 

Nothing is guaranteed in life or science, but we are optimistic that the epalrestat Phase III trial can lead to learning and treatments that will impact the quality of life for your PMM2 CDG family and ultimately for a larger group of related disorders.

Phase III epalrestat trial begins soon…


Dear PMM2-CDG Family,

We’re glad you're interested in learning about the PMM2-CDG clinical trial or our ongoing CDG research.


Please complete this brief form.

Please give us a bit more information so that we can get the ball rolling.


Latest News

Groundbreaking biotech gets closer to saving kids with a rare disease

Word on the street


It's official

Check out the official government
clinical trial page


$2M Grant

Maggie's Pearl wins

"Direct-to-Phase 2" SBIR award

"After several weeks and months the impacts became obvious and the lab results supported the progress and gains we saw. It’s nothing short of a modern miracle – Maggie is better, and I am forever grateful to all who helped us get here.”

Holly Carmichael

Founder and COO

Maggie’s Pearl

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.