DF-003 is the first ALPK1 inhibitor to successfully complete a first-in-human Phase 1 study and has now entered a Phase 1b clinical trial in patients with ROSAH syndrome (NCT06395285), where it is ...
Familial Hypobetalipoproteinemia (FHBL), caused by variants in the apolipoprotein B (APOB) gene, is a rare autosomal co-dominant monogenic disorder characterized by lifelong low plasma levels of total ...
Everyday Health on MSN
Genetics and family risk: What to know if you have hypertrophic cardiomyopathy (HCM)
Learn how hypertrophic cardiomyopathy (HCM) affects families, the importance of genetic testing, and why regular screening is ...
Huntington's disease is caused by an inherited genetic change that causes production of an altered protein. This leads to nerve damage in areas of the brain that control movement and thinking.
Many genetic variants linked to inherited eye disorders rarely lead to disease, meaning up to 2% of people could carry these variants without ever developing vision problems, according to a new s tudy ...
The European Medicines Authority’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization for a new drug to treat hereditary angioedema (HAE).
Kidney stones (nephrolithiasis/urolithiasis) are a clinically significant yet underrecognized complication of autosomal dominant polycystic kidney disease (ADPKD ...
SAN FRANCISCO, Nov. 5, 2025 /PRNewswire/ -- Calico Life Sciences LLC (Calico), a biotechnology organization focused on aging and age-related diseases and founded by Alphabet Inc. and Arthur D.
Goal: Given the systemic nature and cardiovascular risks associated with ADPKD, we sought to investigate the progression of cardiac dysfunction in a mouse model carrying the Pkd1 R3277C (RC) mutation.
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