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 ...
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 ...
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 ...
Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare disease targets.
The FDA has cleared PTC Ther­a­peu­tics’ gene ther­a­py for AADC de­fi­cien­cy, an ul­tra-rare dis­ease that im­pacts the ...
An international, Phase III clinical trial led by investigators at Mass General Brigham could improve the treatment of a rare ...
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 ...