Author Archives: Patricia Inacio PhD

Skin Biopsy May Help Diagnose FAP in Early Stages for Treatment

A skin biopsy may help speed up the diagnosis of familial amyloid polyneuropathy (FAP), including for asymptomatic people with disease-causing mutations who are at higher risk of developing full-blown disease, according to a single-center retrospective study. This would allow patients, especially those with the Val30Met mutation, to receive treatment…

Eplontersen Granted Orphan Drug Status in US for ATTR Amyloidosis

The U.S. Food and Drug Administration (FDA) granted orphan drug status to eplontersen, an investigational therapy that seeks to prevent the buildup of toxic protein deposits in people with transthyretin (ATTR) amyloidosis — a group of disorders that also includes familial amyloid polyneuropathy (FAP). The therapy is being co-developed…

Heart Disease Can Be Prevalent in Men With ATTRv Amyloidosis

Heart involvement is more prevalent among men with hereditary transthyretin (ATTRv) amyloidosis, a group of rare disorders that includes familial amyloid polyneuropathy (FAP), a study reports. Findings suggest that female sex may be protective against heart disease in ATTRv, and that being male may also contribute to disease worsening. The…

Attralus Gets $116M Financing Boost to Develop Imaging Agent

Attralus has raised $116 million to support the clinical development of AT-01, its diagnostic imaging agent for systemic amyloidosis, a group of rare conditions that includes familial amyloid polyneuropathy (FAP). Proceeds from the Series B financing will also be used to develop therapeutic agents for different forms of systemic…

Gene-editing Therapy for ATTR Shows Promise in Preclinical Study

Precision BioScience’s gene-editing therapy for transthyretin amyloidosis (ATTR), a group of disorders that also encompasses familial amyloid polyneuropathy (FAP), shows therapeutic potential in a preclinical study. Data from the study will be presented by Jenny A. Greig, PhD, senior director of the Gene Therapy Program at the Perelman…