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 ...
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 ...
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 ...
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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 ...
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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 ...
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 ...
Family ‘absolutely broken’ as girl, 2, dies three years after brother from same disease
Isabelle Cooper, 2 , died on Thursday September 19, just three years after her brother Alexander died of the same heart ...
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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 ...
Family left ‘absolutely broken’ as girl, 2, dies three years after her brother
A toddler has died after being diagnosed with the same rare genetic disease that killed her three-year-old brother. Isabelle ...
Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C
MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive ...
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.