As of last year, Clementine was just one of 10 people who have been diagnosed with a mutation to the TLR8 gene. She received ...
Are you battling a rare disease or think you might be suffering from one? If so, there are resources to get help from ...
In a new study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs ...
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
The company has decided to advance two new wholly-owned pipeline drugs, AOC 1086 and AOC 1072, targeting two rare genetic ...
As the rollout of its recently launched pulmonary arterial hypertension (PAH) drug Winrevair ramps up, Merck is continuing ...
Over 17 years, researchers and conservationists team to save, then reintroduce, mountain yellow-legged frogs to lakes in ...
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 ...
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