FDA awards orphan drug designation to VPO-227 to treat cholera

Oct. 24, 2022

VPO-227 is a small molecule with a novel mechanism of action, which blocks the cystic fibrosis transmembrane conductance regulator (CFTR) ion channel.

BioWorld Science Gastrointestinal Infection FDA Orphan drug

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BioWorld briefs for Feb. 5, 2025.

MicroRNAs in circulating extracellular vesicles as IPF biomarkers

BioWorld Science

MicroRNAs (miRNAs) are small noncoding RNAs gaining increasing attention due to their crucial role in gene expression regulation and influence in various cellular...

MJFF grant supports Bexorg’s translational biomarker work

BioWorld Science

Bexorg Inc. has been awarded a research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) through the Targets to Therapies initiative to...

REST emerges as biomarker in ALS, knockdown improves ALS symptoms

BioWorld Science

About 10% of amyotrophic lateral sclerosis (ALS) cases result from inherited genetic mutations, with about 20% of them attributed to mutations in the gene...

Illustration of liver being targets with cross-hairs

Study identifies CAND1 as therapeutic target in hepatoblastoma

BioWorld Science

Hepatoblastoma is the most common liver cancer during childhood, with limited therapeutic options in aggressive or relapsed cases. NEDDylation is a...