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 Zevra's Miplyffa for Niemann-Pick Disease Type C — 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 ...
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.
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 ...
Raccoon roundworm infections reported in Los Angeles County, health officials warn
A rare parasitic infection known as raccoon roundworm has been confirmed in Los Angeles. The infection, Baylisascaris ...
A Rare but Deadly Roundworm Spread by Raccoons Is Infecting People in LA
LA public health officials reported this week that two residents have been infected by Baylisascaris procyonis, also known as ...
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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 ...
How is Céline Dion’s health? What she's shared about stiff person syndrome battle
Dion is currently battling a serious neurological condition called stiff person syndrome. Here's what we know.
Bird Flu Symptoms in Humans: How to Protect Yourself and Prevent This Rare But Serious Virus
With cold and flu season approaching, it’s worth knowing about another virus out there—albeit extremely rare: bird flu. Like ...
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Rare Bleeding Disorder, HHT, Treated Successfully with Cancer Drug in Clinical Trial
An NIH-supported clinical trial of pomalidomide in treating hereditary hemorrhagic telangiectasia stopped early due to ...
Raccoon roundworm: What to know about the parasite that infected 2 in California
A rare parasitic infection that is linked to raccoons has been detected in two people in Southern California, the Los Angeles ...