BridgeBio’s Post

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.

In our latest episode of the #OnRarePodcast, we hear from Sean, who shares his diagnosis journey with transthyretin amyloidosis (ATTR), a rare, underdiagnosed, and life-threatening heart disease. Sean describes his initial symptoms, which included carpal tunnel syndrome and numbness in his foot and ankle, the misdiagnosis he experienced, and his eventual diagnosis from his physician. Listen to the episode: https://bit.ly/3VQUPEo 

We are honored to have attended this year’s Iowa Wellstone Dystroglycanopathy Patient & Family Conference hosted by the University of Iowa Roy J. and Lucille A. Carver College of Medicine, where we had the opportunity to meet with individuals and their families and caregivers to learn more about their condition and their unmet needs. During the conference, we shared details of our approach to pursue Accelerated Approval based on using glycosylated alpha dystroglycan as a surrogate endpoint, a muscle biomarker on the causative pathway of limb-girdle muscular dystrophy type 2I/R9. 

Predictive biomarkers of ATTR‐CM can help lead to earlier intervention and allow physicians to better understand and develop a treatment plan for patients with this relentlessly progressive heart disease.

BridgeBio exists to help patients and make a difference in the lives of millions afflicted with genetic conditions. We are hiring Therapeutic Sales Specialists to join us on our mission in the West regions: South California, South Central, Great Plains, Northwest, and Rocky Mountain. Apply today: https://lnkd.in/ep3KmgM6