LIB Therapeutics announced that the US Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA ...

Each episode of this journey through a disease state contains both a physician guide and a downloadable/printable patient ...

Esperion Therapeutics has two approved LDL-C lowering therapies, Nexletol and Nexlizet, but lacks a strong pipeline for future growth.

The cohort's general SOD1 phenotype was characterized by spinal onset without cognitive impairment, with disease progression ...

including heterozygous, and those 10 years or older with homozygous familial hypercholesterolemia (HeFH / HoFH). Lerodalcibep is a novel, adnectin-based, small protein-binding, third-generation PCSK9 ...

Objectives Heterozygous familial hypercholesterolaemia (FH) confers a significant risk for premature cardiovascular disease (CVD). However, the estimated prevalence of FH varies substantially among ...

Purpose: To determine if there is significant symptomatology in women with heterozygous α-galactosidase mutations. Methods: Data from medical records of the 44 heterozygous females followed at ...

These findings indicate that APS therapy might have beneficial effects on DCM. Taking all these findings together, the present study revealed that APS therapy could improve cardiomyopathy in ...

1 Department of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research, University of Tübingen, Tübingen, Germany 2 German Research Center for Neurodegenerative Diseases (DZNE), ...

7 International Research Fellow of Japan Society for the Promotion of Science (Postdoctoral Fellowships for Research in Japan (Standard)), Tokyo, Japan Correspondence to Dr Shinji Saitoh, Department ...

The same novel compound heterozygous PKHD1 variants were detected in both of the fetuses inherited from the parents by whole exome sequencing (WES). Additionally, a minigene experiment was conducted ...