US FDA approves Zevra's treatment for rare genetic disease
The U.S. Food and Drug Administration approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it the ...
Calves to Cure helps fund research for Montana boy's rare disease
The Billings Livestock Commission hosted the sixth annual Calves to Cure to help raise money and awareness for Duchenne ...
Psychology Today2d
How I Survived a Rare Autoimmune Disease
I walked out of my marriage and immediately fell ill. I was ill for two years. At first, I thought it was a cold that would ...
Health officials in L.A. Co. investigating rare disease spread from raccoons to humans
The Los Angeles County Department of Public Health is looking into two human cases of a rare parasitic infection that comes ...
'One small step': Community comes together for Swansea 1-year-old with rare liver disease
A walk-a-thon fundraiser steps off in New Bedford this weekend in support of "Saige the Brave" following the 1-year-old's ...
Woman with rare disease claims she’s now blind and bruised after doctors made her get 3 vaccines
A 23-year-old Florida woman has been temporarily blinded and bruised after allegedly being required to receive multiple ...
STAT14h
FDA approves first drug for rare neuron disease, Niemann-Pick type C
Parents pushed for years for treatments for the neuron disease Niemann-Pick type C. After setbacks, one drug now has FDA ...
Cleveland8h
Rocky River family share story of daughter's rare disease
An Oscar-nominated actor recently spoke exclusively to PEOPLE about his adult son with a rare disease. It inspired a Rocky ...
STAT1d
House panel passes pediatric rare disease bill, setting it up for government funding bill
A House committee on Wednesday passed a bill to renew a program aimed at aiding development of drugs for rare diseases in ...
FierceBiotech2d
FDA scraps adcomm for Applied's rare disease drug ahead of November decision
After pushing back the decision date for Applied Therapeutics’ metabolic disorder drug govorestat, the FDA has now decided ...
fox17online6h
ArtPrize entry highlights rare neurodegenerative disorder
Multiple system atrophy, or MSA, is a rare neurodegenerative disorder with symptoms akin to ALS or Parkinson’s disease in the ...
Targeted Oncology2d
Atypical Chronic Myeloid Leukemia: A Rare Disease With Poor Prognosis
Ongoing research is exploring new molecular targets and targeted therapies for this challenging disease.