Researching Hope for ALG13-CDG
Current Research
The ALG13-CDG Drug Repurposing Project with Perlara has officially launched. The first phase of the project includes the Avatar Characterization & Assay Optimization. The goal of the first phase is to establish a screenable phenotype- a robust and reproducible growth defect. This phenotype serves as a baseline for the screen. During this phase they will assess the growth of ALG13 yeast compared to healthy yeast; ALG13 yeast should grow much slower. Then they will determine the conditions that show the biggest growth difference in a luminescent-based growth assay – temperature and growth time. Lastly, they will optimize these growth conditions in screening plates. This first phase should take about 8-10 weeks, with a goal of completion before mid-August 2025. Once this is complete they will roll right into phase two, Drug Repurposing Screen.
Our Team
We have partnered with the nonprofit organization CDG Care to help raise money for scientific research for ALG13-CDG.
Research Opportunity
The money we raise will help us to partner with the biotech companies like Perlara to find a treatment for ALG13-CDG
Our Mission
To raise money to participate in research opportunities that advance science in the CDG community and focus on finding a cure for ALG13-CDG.
ALG13-CDG Resources
Specialized Medical Care
Dr. Eva Morava, Department of Genetics and Genomic Sciences, Division of Medical Genetics and Genomics at Mt. Sinai
Research Opportunities
Basic Information
Our Community
CDG HERO of the Month
This month, we’re honored to spotlight sweet Rayleeigh, age 2, from the USA, who lives with ALG13-CDG
PKT Hero (Precision Ketogenic Therapy)
Ten year old Siena controls her seizures with only the ketogenic diet.
Leah - ALG13 CDG Hero
Leah has given science more than science could ever give her.
Siena
Her fight and joy for life has given hope to many ALG13-CDG families.
ALG13-CDG News
Saving Siena
Susan Smiley - Height from Ocala Style Magazine helps educate the local community about ALG13-CDG and promote fundraising for ALG13-CDG scientific research.
Glycosylation, CDGs & Role of Sugars Beyond Energy Production
Dr Eva Morava talking in depth about Glycosylation and Congenital Disorders of Glycosylation.
Support CDG & Other Rare Diseases
May 16th is World CDG Awareness Day!
Please join in our efforts to raise CDG Awareness in your Community!
Update to Saving Siena
Ocala gazette has repubished Ocala Style Magazine's "Saving Siena" to include recent updates to fundraiser and research.