Are you battling a rare disease or think you might be suffering from one? If so, there are resources to get help from ...
Shares of Avidity Biosciences RNA rose more than 12% on Wednesday after the company announced that it is expanding its current RNA-based pipeline of rare muscle disorders to explore a new therapeutic ...
In a new study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs ...
The researchers utilized a Fra-2 transgenic mouse model that encompasses many of the features of systemic sclerosis in humans, including spontaneous systemic inflammation and fibrosis in the lungs, ...
Over 17 years, researchers and conservationists team to save, then reintroduce, mountain yellow-legged frogs to lakes in ...
PTC Therapeutics (NASDAQ:PTCT) has received FDA approval for its gene replacement therapy Kebilidi for the treatment of ...
On Wednesday, The Dallas Morning News reported that Dallas restauranteur Al Biernat died at 69 after a battle with ...
Recent outbreaks of Legionnaires’ disease have been linked to U.S. cruise ship hot tubs, according to reports. Private ...
In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs diseases—two rare, often fatal lysosomal storage disorders that cause ...
The FDA has cleared PTC Therapeutics’ gene therapy for AADC deficiency, an ultra-rare disease that impacts the ...
This is evidenced by public statements and congressional testimony by FDA leadership, the Congressional Rare Disease Caucus' communications with FDA as well as published editorials authored by the ...
to use its artificial intelligence to identify novel rare disease indications for a discontinued drug asset. Under the terms of the agreement, Sanofi will provide data related to the unnamed drug ...
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