The Business Journals55m
Durham firm's gene therapy helps patients see first snowflake, star
One of its programs, called called ATSN-101, was recently evaluated in a Phase1/2 clinical trial involving patients with a rare inherited retinal disease — called Leber congenital amaurosis 1, or LCA1 ...
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 ...
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 ...
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 ...
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 ...
Zevra Therapeutics' Drug Becomes First FDA-Approved Treatment For Ultra-Rare Neurodegenerative Disease
The FDA has approved Zevra Therapeutics' Miplyffa (arimoclomol) as the first treatment for neurological symptoms of ...
scrippsnews2h
2 people infected with raccoon roundworm disease. Here's how to avoid exposure
The parasite that causes the disease lives in the intestines of raccoons and can cause serious illness in humans.
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 ...