In a new study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs ...
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RNA Stock Hits Record High on Entering the Cardiac Disease SpaceThe company has decided to advance two new wholly-owned pipeline drugs, AOC 1086 and AOC 1072, targeting two rare genetic ...
On Wednesday, The Dallas Morning News reported that Dallas restauranteur Al Biernat died at 69 after a battle with ...
Alpana Sharma, founder-director of CureSMA, a parent-led advocacy group, urges the government to include medicines for SMA in ...
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 ...
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 ...
The brother of former Channel Nine reporter Hayley Webb has died from a rare brain disease. Lachlan Webb, who is the younger ...
A slow launch for Alzheimer's medicine Leqembi, a lackluster pipeline and a challenging drug launch environment are just a ...
The combined net Q3 2024 revenue of approximately $3.1 million for prescription and non-prescription products, including license revenue, increased approximately 14% versus net Q2 2024 revenue of $2.7 ...
California’s recent decision to create a Rare Disease Advisory Council is a milestone that brings new hope to people living ...
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