Esperion (ESPR) announced that following meetings with the U.S. Food and Drug Administration, it has gained alignment on a regulatory path ...
Confirms Sufficient Data to Complete Phase 2 Clinical Study Enrollment and Advance to Phase 3 Studies in Both Heterozygous and Homozygous Familial Hypercholesterolemia – ...
The ACMG Foundation for Genetic and Genomic Medicine is proud to present the ACMG Foundation David L. Rimoin Inspiring Excellence Award to Ali H. Bereshneh, PhD for his featured platform presentation, ...
Incorporating systems thinking into drug value assessment promises more realistic evaluations of the impact of new therapies, ...
Dosing underway in INLIGHT trial of WVE-007 in obesity with clinical data expected in 2H 2025; enrollment complete in first single dose ...
Verve Therapeutics, Inc. (Nasdaq: VERV) is a clinical-stage company developing a new class of genetic medicines for cardiovascular disease with the potential to transform treatment from chronic ...
Genetic analysis revealed a homozygous pathogenic frameshift variant in BSCL2 (NM_001122955.4:c.974dup; p.Ile326HisfsTer12), classified as pathogenic according to ACMG guidelines, with both parents ...
Having one variant (heterozygous) is less likely to contribute to health issues. Some experts believe that having two mutations (homozygous) may lead to more serious problems. There are two ...
Histone H3 lysine 4 methylation is one of the most abundant epigenetic modifications, which has been recently linked to vascular remodeling in pulmonary hypertension (PH). SET1/MLL methyltransferase ...
Proper connectivity of the nervous system requires temporal and spatial control of axon guidance signaling. As commissural axons navigate across the CNS midline, ROBO-mediated repulsion has ...