Confirms Sufficient Data to Complete Phase 2 Clinical Study Enrollment and Advance to Phase 3 Studies in Both Heterozygous and Homozygous Familial Hypercholesterolemia – ...

Esperion (ESPR) announced that following meetings with the U.S. Food and Drug Administration, it has gained alignment on a regulatory path ...

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, ...

Even as Novartis takes home a partial win in court tied to its key blockbuster Entresto, the Swiss drugmaker continues to ...

Investigators advised clinicians of the potential for dystrophies that mimic age-related macular degeneration and other ...

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Huntington's disease is a rare genetic, autosomal dominant, neurodegenerative disease, caused by CAG repeat expansions in the HTT gene, that is polymorphic on the healthy allele and contains more than ...

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 ...

The table is a review of notable updates that occurred in February 2025 for investigational products in development.

Incorporating systems thinking into drug value assessment promises more realistic evaluations of the impact of new therapies, ...