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 ...
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AIRNA Hits $155M Series B Haul To Advance RNA Editor for Rare Genetic Disease
AIRNA’s lead candidate AIR-001 works by correcting the most common pathologic mutation driving the rare disease alpha-1 ...
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 ...
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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 ...
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Buddhist devotee embraces Catholicism after healing 'miracle'
Sister Rasika, who had supported Ramyalatha for years, played a pivotal role in her spiritual journey. “You should pray ...
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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 ...