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 approves first treatment for rare genetic disorder Prader-Willi syndrome
The U.S. Food and Drug Administration on Wednesday approved Soleno Therapeutics' drug to treat a rare genetic disorder, ...
Dallas scientists granted $420K to test gene therapy for rare neurological disease
Childhood-onset striatonigral neurodegeneration robs children of the ability to walk and talk by age five. Researchers at ...
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, ...
This internationally recognized NJ doctor is on the frontlines battling rare diseases
A rare disease in the US is one in 1,500, or fewer than 200,000 people, and an ultra-rare disease is one in 50,000 or fewer ...
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 ...
AsiaOne2d
'My skin is nice': Raising awareness for children with rare skin conditions
"Why did my classmate say that my skin is yucky?" asked five-year-old Sophie Goh. It may seem like an innocent question, but ...
The Thaiger49m
No boots for blisters: Thai military service exemptions updated
Thailand’s Defence Ministry added three unusual medical conditions to the exemption list for compulsory military service.
Rolling Out5d
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 ...
STAT5d
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 ...
BioPharma Dive5d
Epicrispr banks $68M to test epigenetic editing on rare muscle disease
The startup is using CRISPR tools to stop errant expression of a gene linked to facioscapulohumeral muscular dystrophy, a ...
USA Today21d
What is PolG? The rare genetic disease that ended a young prince's life
Prince Frederik of Luxembourg died from a genetic disease called PolG, but experts hope his passing could bring more awareness to the rare condition that impacted his life. Prince Frederik ...