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 ...
New York Post1h
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 ...
FiercePharma1h
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 ...
Zevra Therapeutics' Drug Becomes First FDA-Approved Treatment For Ultra-Rare Neurodegenerative Disease
Miplyffa (arimoclomol), an oral medication for Neimann-Pick disease, type C (NPC). Miplyffa, in combination with Johnson & ...
Business Wire11h
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 ...
STAT19h
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 ...
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 ...
MedPage Today19h
First Drug Approved for Niemann-Pick Disease Type C
Opens in a new tab or window The FDA on Friday approved arimoclomol (Miplyffa) as the first treatment specifically for the ...
STAT22h
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 ...
Buy Rating Justified by Azitra Inc’s Advances in Rare Skin Disease Treatments and Financial Stability
Jason McCarthy, an analyst from Maxim Group, reiterated the Buy rating on Azitra Inc (AZTR – Research Report). The associated price ...
KTVQ Billings, MT on MSN17h
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 ...