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These beautiful children are suffering from a rare disease caused by loss of function of the FAM177A1 gene. Disruptions of the FAM177A1 gene cause intellectual disability, seizures, autism, progressive motor decline and other debilitating issues.
There are currently no disease-specific treatments.
The FAM177A1 Research Fund is a family-led patient advocacy organization dedicated to improving the lives of everyone impacted by FAM177A1-related Neurodevelopmental Disorder. We provide hope to affected families while focusing the global research community on FAM177A1 to develop life-transformative treatments for FAM177A1 Disorder.
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We know the gene. We have the team. Let's do this!
SYMPTOMS/PHENOTYPE of FAM177A1 Associated Disease
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Intellectual disability/global developmental delay
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Autism
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Macrocephaly/Large head size
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Seizures
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Sleep disturbance
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Unusual gait or walking pattern
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Congenital cataracts
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Muscle tone issues
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Immune issues such as arthritis and frequent infections
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Upslanted palpebral fissures
ENGAGE
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Does your child have FAM177A1 Associated Disease?
You are not alone!
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Do you know a child that is similar to our sweet zebras? Check their symptoms and get free genetic testing.
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