Maggie's Pearl

Clinical-stage biotech company developing epalrestat as the first targeted therapy for PMM2-CDG and other Congenital Disorders of Glycosylation

[Maggie's Pearl @epalrestat]

Phase III clinical trial of pediatric subjects with PMM2-CDG begins crossover to open label epalrestat https://t.co/BAQULDrozi

[EveryLife Foundation @EveryLifeOrg]

Wrapping up an enlightening Rare Disease Caucus briefing on "Novel Approaches to Drug Development for Small Rare Disease Populations—and the Policies that Can Drive Them." A huge thank you to our insightful speakers (Adora Ndu, @eperlste , Dominique Pichard, Christine Waggoner, Annie Kennedy) Let's keep the momentum going and register for our #ScientificWorkshop2024 https://t.co/OsDyftl9BS

[Akhilesh Pandey Lab @Lab_Pandey]

Our collaborative work with the Tamas Kozicz and @EvaMorava team describing a new cortical brain organoid model of PMM2 Congenital Disorder of Glycosylation led by @Radenkovic_S and @budhraja_rohit has just been published in @CellReports #glycotime @FrontierCDG

[Sylvia Radenkovic @Radenkovic_S]

In the latest collaboration efforts led by @kilroykishore and @Lab_Pandey , we identified complement4 as a novel biomarker for PMM2 deficiency! @FrontierCDG @CdgHub

[Ethan Perlstein 1-to-N @eperlste]

Our pivotal Phase 3 trial advances to open label!

[Mayo Clinic Ventures @MayoInvents]

A collaboration between @PerlaraPBC, Maggie’s Cure, and @MayoClinic has announced significant progress in their Phase III trial of epalrestat, an experimental drug meant to combat a rare genetic disease. Read more here:
https://t.co/a3bpw1uRZa

#MayoClinic #Innovation #Genetics

[World CDG Organization @worldCDG]

🎉 That‘s a wrap! Thank you everyone!
🤝 Together, we‘ve expanded our knowledge, challenged preconceptions, and strengthened the bonds within our community.
Let‘s keep pushing the boundaries and advocating for the CDG community.

#6thWorldConferenceonCDG

[Mayo Clinic Ventures @MayoInvents]

This impactful work was enabled by a biotech #collaboration between @epalrestat and @MayoClinic.

[FCDGC @FrontierCDG]

Check out the tweetorial of our #FCDGC investigators at @rarediseasesnet for insights into new potential treatment for #PMM2-#CDG, now a phase III clinical trial.

[Sylvia Radenkovic @Radenkovic_S]

After 4 years our article is finally out! Many thanks to supervisors @EvaMorava and @VIBMetaboCoreLV ! We investigate the link between abnormal glycosylation and polyol metabolism, and show targeting polyol metbolism results in improved glycosylation in PMM2-CDG! Check it out👇🏻

[Nature Biotechnology @NatureBiotech]

From made-to-order genetic therapies to model organisms engineered to be ‘patient avatars’, the technology exists right now to save patients with rare diseases https://t.co/2ZTKacIdvg

[Nature Biotechnology @NatureBiotech]

Personalized medicine is having its day
From made-to-order genetic therapies to model organisms engineered to be ‘patient avatars’, the technology exists right now to save patients with rare diseases https://t.co/2ZTKacIdvg

[Mayo Clinic Ventures @MayoInvents]

.@MayoClinic entered into a know-how agreement with @epalrestat in 2021 to develop the first platform therapy for a group of related genetic inborn errors of metabolism called PMM2-CDG. Today that therapy is having real impact! Read more: https://t.co/pzvlo2QJL7 #RareDiseaseDay

[Mayo Clinic Center for Individualized Medicine @MayoClinicCIM]

Months after Maggie started taking an experimental drug for her ultra-rare genetic disease, she traded in her wheelchair for a walker. The 9-year-old also became better at feeding herself, her speech improved and she started coloring in the lines. https://t.co/9gzDqvc9uQ

[Rare Diseases Clinical Research Network @rarediseasesnet]

Meet Maggie Carmichael, a patient with a #CongenitalDisorderOfGlycosylation who is receiving a new treatment pioneered by Eva Morava-Kozicz, MD, PhD, principal investigator of @FrontierCDG, and collaborators: https://t.co/81xuyWWGL2

#RareDiseaseDay #RDDNIH

[Mayo Clinic Center for Individualized Medicine @MayoClinicCIM]

A Mayo Clinic discovery has led to a life-changing treatment for young girl with an ultra-rare disease. Read more: https://t.co/VU8ciwYKJQ #RareDiseaseDay

[Maggie's Pearl @epalrestat]

Maggie’s Pearl Initiates Phase III Clinical Trial for Treatment of Patients with PMM2-CDG https://t.co/YDf3wuSHX6

[Maggie's Pearl @epalrestat]

To learn more, please reach out to the Clinical Research Coordinator at Mayo Clinic who is managing study enrollment: [email protected], 507-266-9619

[Maggie's Pearl @epalrestat]

Maggie’s Pearl Initiates Phase III Clinical Trial for Treatment of Patients with PMM2-CDG https://t.co/YDf3wuSHX6

[Maggie's Pearl @epalrestat]

The 40-patient placebo-controlled Phase 3 study of epalrestat for the treatment of PMM2-CDG is actively recruiting https://t.co/864efXdkBG