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 ...
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 ...
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 ...
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 ...
Voucher program notches win as clock ticks
The FDA’s priority review voucher program for rare pediatric diseases is set to lapse on Sept. 30 without congressional intervention.
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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 ...
KTVQ Billings, MT on MSN19h
Calves to Cure helps fund research for rare disease battled by Worden boy
This U.S. Army film tells the story of a Japanese woman who marries an American serviceman and goes with him to the United ...
Science Daily1h
Unlocking the potential of patient-derived organoids for personalized sarcoma treatment
Investigators developed the largest collection of sarcoma patient-derived organoids to date that can help improve the understanding of the disease and better identify therapies that are most likely to ...
Vaccine horror as woman, 23, left ‘temporarily blind with agonising bruises engulfing her face’
A WOMAN claims she was left temporarily blind with agonising purple bruises spreading across her face after having three ...
FDA Approves Zevra's Miplyffa for Niemann-Pick Disease Type C — 2nd Update
Zevra Therapeutics has won Food and Drug Administration approval of Miplyffa for Niemann-Pick disease type C, or NPC, making it the first drug cleared in the U.S. for the rare neurodegenerative ...