Findings suggest that urinary ceramide levels could serve as an early biomarker for acute kidney injury, helping doctors identify patients at risk.
Isle of Wight County Press on MSN
Family's Christmas hamper aim for hospital's children's ward
The parents of an Isle of Wight child with a rare mitochondrial condition are asking for donations to provide Christmas ...
Around 25 million Americans have a rare genetic disease, and many of them struggle with not only a lack of effective ...
News-Medical.Net on MSN
Multiple protein forms from a single gene offer fresh insight into rare disease mechanisms
Iain Cheeseman and colleagues reveal the underappreciated role of single genes producing multiple proteins in atypical ...
Kygevvi (doxecitine and doxribtimine) earned FDA approval as the first treatment for thymidine kinase 2 deficiency, supported by data from a Phase II trial and multiple retrospective studies showing ...
The First And Only Treatment For Adults And Children Living With Thymidine Kinase 2 Deficiency (Tk2d). <li /> Approved indication: KYGEVVITM (doxecitine and doxribtimine) powder for oral solution (2 ...
A widely prescribed diabetes drug may be sabotaging one of the most trusted strategies for preventing the disease: exercise.
The FDA declined to consider elamipretide for full approval in 2021 and rejected the company's application in May of 2025 ...
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