BridgeBio

#DYK that low levels of serum TTR in ATTR-CM is an indicator of destabilization of TTR, which is correlated with earlier disease onset and increased severity? We're proud to be advancing research for the unmet needs of ATTR-CM patients.

In our latest episode of our OnRare Podcast, we hear from Misty, who shares her powerful journey living with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), an inherited condition that causes progressive deterioration of the muscles. After years of being dismissed by medical professionals, she finally received her diagnosis—only after her twin sisters were given the same diagnosis. Listen to her inspirational story here: https://bit.ly/480Bfew??

As part of Dwarfism Awareness Month, we're highlighting the unique voices of individuals who remind us that the skeletal dysplasia community offers invaluable perspectives of society through their diverse and rich experiences. Visit MyAchonJourney.com to hear more of these voices, explore resources, and learn more about advocacy organizations that may help you connect with others in this vibrant community. #AmplifyCommunityVoices

We're thrilled to have recently shared details about FORTIFY, our Phase 3 study for individuals with limb-girdle muscular dystrophy type 2I/R9 at the World Muscle Society Congress. Topline data from an interim analysis after 12 months post-dosing is expected to be available in 2025. Learn more: https://bit.ly/4gDBd07

In recognition of Dwarfism Awareness Month, we're honored to share the story of Alex, Colin, their daughter Stella, and their two-year-old son Hudson, who lives with hypochondroplasia. In this heartfelt video, Alex and Colin reflect on learning about Hudson's diagnosis during pregnancy, the incredible support they received from their community, and the joy they find in watching him grow into a happy, thriving child. Thank you to Hudson's family for sharing your journey. #AmplifyCommunityVoices

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.

Sean, who lives with ATTR-CM, recently joined our #OnRarePodcast and spoke about the many symptoms he experienced before receiving a diagnosis. He noted that finding out about his condition was a tremendous sense of relief, but a big change for him that he chose to look at positively. Learn more about Sean’s story: https://bit.ly/3VQUPEo

We’re thrilled to share our outcomes data through 42 months from our long-term open-label extension of ATTRibute-CM, our Phase 3 study of acoramidis in ATTR-CM, in a featured science oral presentation at the 2024 American Heart Association Scientific Sessions. In addition to the presentation, BridgeBio was selected to share three posters in moderated poster sessions on ATTR-CM. Learn more:?https://bit.ly/4eNw9V8

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.

We appreciate the opportunity to meet with experts and advocates in the hypoparathyroidism community at this year’s American Society for Bone and Mineral Research (ASBMR) Annual Meeting. We look forward to applying our learnings in the future. #ASBMR2024