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, ...
How Denali Therapeutics‘ breakthrough may help another firm win approval of a rare disease drug
Scientifically, Denali's milestone filing is important because it shows the potential of the company's enzyme transport ...
EXCLUSIVE: How 1 dad used AI to advance finding a treatment for his son’s rare disease
AI helped Thomas Wagner understand Alexander disease, find connection with other conditions and discover researchers who ...
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)
Cerebrocostomandibular syndrome is a rare genetic disorder characterized by a combination of abnormalities affecting the ...
3rd Annual TARS Awareness Texas 5K to highlight challenges and hope for those with the rare condition
CORPUS CHRISTI, Texas — Local non-profit, TARS Awareness Texas, is asking you to join them at their 3rd annual 5K run and ...
AsiaOne3d
'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 ...
Why Ultragenyx Pharmaceutical (RARE) Is Among the Best Mid Cap Biotech Stocks to Buy
We recently published a list of the 10 Best Mid Cap Biotech Stocks to Buy. In this article, we are going to take a look at ...
Rolling Out7d
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 ...
Early-onset Alzheimer’s: Drug shows promise in slowing the disease
Early-onset Alzheimer’s disease progresses more rapidly and often strikes people in the prime of their lives. A new treatment ...