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 ...
Opinion: A $22 billion life sciences engine vs. Washington red tape
Utah's life sciences sector is a significant economic powerhouse, but the misguided the Medication Affordability and Patent ...
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 ...
FDA Approves First Treatment for Niemann-Pick Disease, Type C
Today, the U.S. Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC).
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 ...
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How you can help Miracle and other sickle cell patients at blood drive
Miracle was born with sickle cell disease, a condition where a person’s red blood cells become rigid, blocking blood flow to the body.
Ipsen’s Iqirvo® (elafibranor) approved in the European Union as first new treatment for primary biliary cholangitis in nearly a decade
European Commission grants conditional marketing authorization for Iqirvo® (elafibranor), first-in-class new treatment for primary biliary ...
Wastewater monitoring can detect foodborne illness, researchers find
First used in the 1940s to monitor for polio, wastewater surveillance proved such a powerful disease monitoring tool that the ...
These Women Knew Their Genes Were Betraying Them
Jenssen guessed then that two of her daughters, the twins, had a Duchenne mutation, and she knew in her heart that, for at ...