If you haven't heard of Rett syndrome, it's probably because it's very rare. Affecting an estimated one in 12,000 baby girls ...
The U.S. Food and Drug Administration said on Tuesday it has approved SpringWorks Therapeutics' drug to treat a type of rare ...
The Food and Drug Administration (FDA) approved a new drug to treat a disorder causing the growth of non-cancerous tumors on ...
Researchers at Washington University School of Medicine in St. Louis have conducted a longitudinal study on an individual ...
More than 1,000 genes may serve as possible treatment targets for individuals with kidney disease, according to a new study, ...
Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...
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Adial Pharmaceuticals, Inc. (NASDAQ:ADIL) announced patent number 12,221,654 was issued on February 11, 2025, by the United ...
A groundbreaking study has identified how previously mysterious mutations in non-coding DNA contribute to kidney disease, ...
People of African descent often have non-communicable and infectious diseases at the same time. Research needs to be done ...
A groundbreaking study reveals how a rare combination of genetics, proteomics, and environmental factors may hold the key to delaying or preventing Alzheimer's, challenging long-held assumptions about ...