US FDA approves Zevra's treatment for rare genetic disease
The U.S. Food and Drug Administration has approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it ...
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 ...
Commentary: Congress has just weeks to help children with rare diseases
A rare-disease federal incentive program critical to biotech R&D is in jeopardy, writes Leslie Williams, president and CEO of ...
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 ...
New York Post3h
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 ...
9-case cluster of rare pneumonia being investigated by Gaston County health officials
Public health officials are investigating a cluster of nine confirmed cases of Legionnaires’ disease among people who ...
‘Survivor 47’ preview: Terran ‘TK’ Foster on facing death’s door because of ‘pretty rare kidney disease’ [WATCH]
Survivor 47” fans, it’s time to meet Terran “TK” Foster, a 31-year-old athlete marketing manager from Upper Marlboro, ...
FiercePharma3h
After prior snub, Zevra clinches FDA approval for rare lysosomal storage disorder drug Miplyffa
For Zevra Therapeutics and its rare neurodegenerative disease med arimoclomol, the second time’s the charm. | The ...
STAT21h
FDA approves first drug for rare neuron disease, Niemann-Pick type C
The Food and Drug Administration on Friday approved the first treatment for Niemann-Pick disease, type C, the culmination of ...
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 ...
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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 ...