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Parents of Boy with Brain Disorder Raise Money for Gene Therapy Clinical Trial
Bozeman family faces the unthinkable as child battles rare genetic disease
Severe combined immunodeficiency (SCID)
They Created a Drug for Susannah. What About Millions of Other Patients? - The New York Times
A quest for Quincy: Gene therapies come of age for some forms of autism, Spectrum
Lorenzo's Oil Could Not Cure Lorenzo, But Newborn Screening Is Expected To Save Others From His Fate
Bremerton mom takes to TikTok to raise awareness about her son's terminal disease
Probing the Cause of a Rare Disease Graduate School of Biomedical Sciences
Genetics Team Serves as Go-to for Mom of Son With Extremely Rare Disorder - Stanford Medicine Children's Health BlogHealthier, Happy Lives Blog
Genetic Disorders: MedlinePlus
Penn rare-disease scientist studies daughter's epilepsy, wins $25 million grant
My Son Has a Rare Terminal Genetic Disorder Called TBCD
Home - Alpha-1 Foundation
Toronto boy whose parents raised $3M to treat his rare genetic disorder starts clinical trial