About Finding a Cure for ALG13-CDG
We are a community of families brought together by a shared similar experience of raising children with an extremely rare genetic disorder known as ALG13-CDG, which is one type of a group of metabolic diseases called Congenital Disorders of Glycosylation (CDG). ALG13-CDG is caused when a mutation either arises spontaneously in an individual's ALG13 gene (located on the X chromosome) or it has been inherited.
There are fewer than 100 known children affected globally, but as genetic testing becomes widespread more cases are identified each year. Nearly all children with ALG13-CDG experience neurological and developmental delays, severe muscle tone disorders, gastrointestinal problems, cortical visual impairment, and seizures. Sadly, we have lost many wonderful children to this medically complex disorder over the years, but every day, we try our best to provide them with the love and support they need to thrive.
There is no cure for ALG13-CDG, so we’ve taken matters into our own hands, and we are collaborating with Perlara, a biotech company, to embark on a drug repurposing project to find a potential treatment to improve our children’s quality of life. Our initial goal is to raised $150,000 which will allow us to complete the first three phases of the drug repurposing project (i.e., optimize yeast avatar, drug repurposing screens, and confirm findings) Our long-term fundraising goal is to continue to raise funds for future phases of the drug repurposing project (e.g., clinical trials) and to participate in any scientific research opportunities that arise for ALG13-CDG.
This multi-phase project is just the start of our ambitious goal of finding a cure for ALG13-CDG, so we’re asking you to join us on our journey to help raise funds and awareness, and hopefully, one day we can reach this incredible goal together!
Researching Hope for ALG13-CDG
Our Mission
Research Opportunity
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.
Our Team
Research Opportunity
Our Mission
We have partnered with the nonprofit organization CDG Care to help raise money for scientific research for ALG13-CDG.
Research Opportunity
Research Opportunity
Research Opportunity
The money we raise will help us to partner with the biotech companies like Perlara to find a treatment 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
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Finding a Cure for ALG13-CDG
