Scientists granted $420,000 to test gene therapy for rare neurological disease
Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by ...
US FDA greenlights first-ever treatment for rare genetic disorder
The U.S. Food and Drug Administration approved Soleno Therapeutics' drug to treat a rare genetic disorder on Wednesday, ...
Her Baby Had to Be Revived 8 Times in the NICU. Now She's Talking About His Rare Condition to Help Moms Like Her (Exclusive)
Audrey Kandil tells PEOPLE her son, Rami, was born with cerebrocostomandibular syndrome, which is a rare genetic disorder ...
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Soleno Therapeutics’ ‘life-changing’ rare-disease drug approved
The FDA Wednesday approved the first drug to treat people with Prader-Willi syndrome, a rare genetic disease that causes an ...
Parents' lives 'turned upside down' as baby boy given very rare diagnosis
A couple have opened up about the heartbreaking moment they learned their baby boy was blind. Albie Moore was born in October ...
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‘Every day counts’ – call to add screening for rare muscle-wasting condition to newborns’ ‘heel-prick’ test
A majority of people have supported the urgent implementation of early screening for a genetic disease that causes severe ...
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Promising drug cuts Alzheimer’s risk by half in rare cases
Research shows experimental anti-amyloid drug reduced cognitive symptom risk by 50% in people with rare genetic mutations ...
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Inherited Metabolic Disorders
a specialist in medical genetics at the Cleveland Clinic Center for Personalized Genetic Healthcare in Cleveland, OH. Each of these inherited metabolic disorders is individually very rare.
Her son has a rare genetic disorder, so this Connecticut mom is taking fight for funding to D.C.
Families in Connecticut and beyond are looking for answers when it comes to treating Angelman Syndrome, but fear funding ...