Mackenzie's Mission

In the latest episode of our #OnRarePodcast, Jonathan Fox, M.D., Ph.D., President and CMO of BridgeBio Cardiorenal, explains the differences of the ATTR T80 variant. Previously, we learned that transthyretin amyloidosis (ATTR) is caused by the dissociation of a protein called transthyretin, or TTR, which changes its shape and forms into fibrous clumps. These clumps of misshapen protein are deposited into various organs and peripheral nerves, including the heart, which can cause them to function abnormally. Jonathan explains the possible origin of the T80 variant and how the T80 variant symptoms can differ from ATTR-CM. Listen to the full episode here: https://bit.ly/3VQUPEo 

Talking to your doctor is an important step in the diagnostic journey, especially if your symptoms are similar to those of heart failure. The earlier the diagnosis, the more of a chance for better clinical outcomes in patients with ATTR-CM.

⏰ BREAKING: Today, the EveryLife Foundation is thrilled to learn of FDA’s establishment of the Rare Disease Innovation Hub, an intercenter collaboration to address common scientific, clinical and policy issues related to rare disease product development. In 2018, our community began advocating for the formation of a rare disease intercenter within FDA alongside our broad coalition partners and Congressional rare disease leaders. All along, this effort has been informed by Congressional, scientific, and policy leaders – and championed by thousands of rare disease community members. But most importantly, this effort has been informed by the experiences of our rare disease community. A community with unparalleled unmet needs and whose engagement has led us to this moment. We are grateful to FDA for recognizing the opportunity to enhance and streamline their existing processes. As a community, we are eager to support implementation and inform the strategic direction of the Rare Disease Innovation Hub. To read the EveryLife Foundation's full statement visit: https://bit.ly/3xRIWGD

Recognizing the early signs of AL #amyloidosis is crucial, leading to earlier diagnosis and improved treatment outcomes for patients. At Prothena, we’re committed to developing novel and transformative medicines to create a better future for people in critical need. Learn more: https://bit.ly/45sSPGQ