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amyloidosis clinical trials

FDA Drug Approval Process

Drugs are the lifeblood of patient treatments, and the development of new drugs is critical. Overseen by the FDA (U.S. Food and Drug Administration), in this blog we outline the phases and 12 steps required to develop and approve drugs.

CRISPR/Cas9 – Editing the Code of Life

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.

Amyloidosis By The Numbers

An inside look into the state of amyloidosis today, based on 575 online responses. Read about patient demographics, symptoms, getting to a diagnosis through the physician community, treatments, and how life has changed post-treatment.

Clinical Trials 101

Clinical research is simply medical research involving people. There are two types, clinical studies (aka observational studies) and clinical trials. In this blog, we explore clinical trials and the basics of what you need to know.