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Currently, MBX-8025 is being evaluated in high unmet need and orphan diseases. HoFH is characterized by loss-of-function mutations in both alleles of the LDL receptor (LDL-R) gene and

The approval is based on data from a pivotal multicenter, randomized, controlled, Phase III trial designed to compare the safety and immunogenicity of Quadracel vaccine with DAPTACEL (DTaP)

Currently, the company is conducting a Phase II trial evaluating the safety and efficacy of ganaxolone as adjunctive therapy for uncontrolled seizures in PCDH19 female pediatric epilepsy, and

In July 2014, the company has secured orphan drug designation from the US Food and Drug Administration (FDA) for RG-012 to treat Alport syndrome. Currently, the company is

As part of the deal, AstraZeneca will provide funding for a team of investigators at Harvard and will establish an in-house team in Sweden which will work on

AG-348 is a first-in-class, orally available activator of pyruvate kinase-R (PKR) enzymes and the medicine met its primary endpoints in two Phase I healthy volunteer trials including a

NuThrax is a combination of BioThrax (Anthrax Vaccine Adsorbed) and the immunostimulatory oligodeoxynucleotide compound CPG 7909. The contract includes a 30-month base period of performance to develop NuThrax

In the trial, NGM282 showed statistically significant, dose-dependent and clinically meaningful reductions in alkaline phosphatase (ALP), a primary indicator of disease activity, as well as substantial reductions in

The probiotic strains used in HLC Baby B and HLC Baby F probiotic formulas were evaluated in a recent clinical trial, in which they were found to be