Etiqueta de tipo de amiloidosis: Amiloidosis ATTR hereditaria

La amiloidosis ATTR hereditaria es una enfermedad hereditaria en la que el cuerpo produce una forma mutante de una proteína llamada "transtiretina". La transtiretina se abrevia como "TTR" y es la razón por la cual esta enfermedad se llama amiloidosis ATTR.

A Multiple-Dose Study in Subjects With Cardiac AL and ATTR amyloidosis to Assess the Safety, Tolerability, and Pharmacokinetics of AT-02 followed by a 2 year open label study in patients with cardiac AL and ATTR

AT-02 is a monoclonal antibody that is being developed to treat systemic amyloidosis by attaching to and stimulating the removal of amyloid fibrils. The goal of this study is to evaluate the safety and tolerability of multiple doses of AT-02 in patients with Cardiac AL and ATTR amyloidosis, and to assess clinical activity. Patients who participate in the Phase 1 part of the study will receive 2 infusions of AT-02 over 4 weeks, [SG1] with a follow-up at week 8. All participants will receive AT-02.