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This status is aimed at facilitating the development and speeding up the review process for drugs that address serious conditions and fill unmet medical needs. Biogen development head

This announcement follows the US Food and Drug Administration’s (FDA) orphan drug designation granted to deucrictibant for the same indication in March 2022. According to the company, this

This new option is intended for patients whose COPD is not sufficiently controlled with existing treatment. COPD is a chronic respiratory disease that progressively impairs lung function and

Acoramidis is a selective small molecule that is orally administered. ATTR-CM is a progressive, restrictive cardiomyopathy that leads to heart failure. BridgeBio Cardiorenal chief medical officer Dr Jonathan

The FDA’s approval adds to its five prior approvals and is based on positive outcomes from the CABINET Phase III trial. CABINET is a multicentre, randomised, double-blinded, placebo-controlled

This condition, caused by genetic mutations in the LEPR gene, leads to disrupted melanocortin-4 receptor (MC4R) signalling, resulting in extreme, constant hunger and severe early-onset obesity. PL7737, an

The treatment, designed to be administered either as an injectable or an oral medication, utilises the regulation of the brain’s nitric oxide (NO) levels to influence neurological function.

The centre will focus on the early detection of diseases and design treatments for improved patient outcomes using precision medicine. Metabolomics, the study of small molecules or metabolites,

The therapy claims to be the only oral inhibitor targeting the alternative complement pathway, specifically addressing what is believed to be the root cause of the disease. Usually