Amyloidosis is a “group of diseases” that have the common feature where abnormal proteins (or in some cases normal proteins) behave abnormally, and the breakdown product of these proteins fold upon themselves, creating amyloid” fibrils.” Amyloid fibrils may affect only a single organ or, often, are spread throughout the body. They can affect different organs in different people, and there are different types of amyloid proteins, making this complex disease often elusive to diagnose. Severe amyloidosis can result in life-threatening damage to these organs or even failure. This overview offers a brief summary of this complex disease.
What Is Amyloidosis
In this special video, hear world-renowned expert Dr. Mathew S. Maurer and his patient John Basdavanos presenting to a group of medical students. Dr. Maurer provides a brief overview of Wild-Type Amyloidosis (ATTRwt), while John provides the patient perspective. Together, these insights offer a compelling story about battling a life threatening disease.
The scientific world is abuzz … a Nobel Prize-winning technology called CRISPR/Cas9 can now edit our DNA. This programmable gene-editing technology, which is efficient, precise, and scalable, has inspired a gold rush of countless applications in medicine, agriculture and basic science. In this blog we invite you to read more about this ground-breaking advance and how, with one course of treatment, it could potentially cure hereditary transthyretin (hATTR / ATTRv) amyloidosis.