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 ...
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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 ...
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A rare disease program, portrayed as both lifeline and pharma handout, is set to expire
Advocates for people with rare diseases say a government voucher program has been crucial to pursuing treatments. That ...
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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 ...
Bored Panda on MSN1d
Dog Has A Rare Disease That Makes Her Body Frozen, Surprises Everyone And Recovers Against All Odds
For example, this one vet chose to care for this pitbull puppy despite the fact that she had a rare and very serious disease ...
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Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy
Neu refix granted orphan disease designation (ODD) & rare pediatric disease designation (RPDD) by US FDA for treatment of ...
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Atypical Chronic Myeloid Leukemia: A Rare Disease With Poor Prognosis
Ongoing research is exploring new molecular targets and targeted therapies for this challenging disease.
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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 ...
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Calves to Cure helps fund research for Worden boy's rare disease
The Billings Livestock Commission hosted the sixth annual Calves to Cure on Thursday, an event to help raise money and ...
Golf tournament to raise money for research on rare disease
The Little Giants Foundation will hold its first-ever golf tournament to raise money for research into a rare, life-limiting ...