BridgeBio

The progression of ATTR‑CM lessens people’s strength, mobility, and independence. While advancements have been made, there is still work to fulfill unmet needs for patients.

Yesterday, TSC Alliance staff members met with our partners at BridgeBio to share community stories and discuss the history, work, and initiatives of the TSC Alliance. This meeting fostered a deeper understanding of BridgeBio's efforts to develop meaningful new treatments for the TSC community.

We were excited to host Congresswoman Deborah Ross at our BridgeBio Gene Therapy labs today in North Carolina. We appreciate the opportunity to share the research and development work we’re doing in gene therapy and to discuss policies that will benefit patients with rare diseases.

We partnered with the community to develop a helpful guide for parents raising a child with achondroplasia. Coming soon. Sign up to get notified: https://lnkd.in/eDRFmjcC #achondroplasia #achon #skeletaldysplasia  

We want to express our gratitude to Sean, who candidly shared his experience about living with transthyretin amyloidosis (ATTR), a rare, underdiagnosed, & life-threatening heart disease, on our recent episode of the #OnRarePodcast. We appreciate your openness when describing how your condition has impacted you and the positive changes you have made to embrace it. Thank you, Sean, for raising awareness for the ATTR community and providing hope to others with the condition. https://bit.ly/3VQUPEo 

Destabilized TTR proteins can aggregate and form toxic amyloid fibrils in the heart and other areas in the body, causing signs and symptoms associated with ATTR-CM.

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.

Putting patients first is a value that BridgeBio practices daily. Part of that is hearing from the people impacted by the conditions we seek to serve. We were fortunate to host and hear from a group of young people living with limb-girdle muscular dystrophy (LGMD), an inherited condition that causes progressive muscle deterioration. It was an incredible opportunity to gain insight from their perspective of how living with LGMD affects their lives. Through these learnings, we can better understand the unmet needs of people with this condition. We are truly grateful for their time and willingness to share their personal stories!

The most beneficial TTR gene variant identified to date is T119M, which has shown to offer protection against the development of ATTR‑CM even when coinherited with variants associated with poor outcomes.