This Medudy course is a video tutorial for physicians on the topic "Patisiran in transthyretin amyloidosis with cardiomyopathy". The format used here is a so-called "Journal Club Summary", in which we summarize the latest and most clinically relevant studies for you.
Transthyretin amyloidosis (ATTR) is a progressive disease caused by misfolded transthyretin
misfolded transthyretin that accumulates as ATTR amyloid fibrils in multiple organs.
The accumulation of ATTR amyloid deposits in the heart often manifests as progressive cardiomyopathy.
Patisiran, an RNA interference therapeutic, inhibits the production of hepatic transthyretin.
The efficacy and safety of patisiran in the treatment of transthyretin amyloidosis with cardiomyopathy is the subject of ongoing studies.
Note: All "speakers" appearing in the Medudy videos are AI-based avatars used to improve the didactics of the videos. The people on whom the avatars are based have explicitly consented to their use. All content is based on high-quality scientific sources and is created and reviewed by the Medudy medical team.
At the end of this training you will know
- the clinical background and relevance of the research question under investigation
- the study design and methodology of the publication to answer the research question
- the results and key takeaways of the publication
- the conclusion of the publication and possible limitations
