Fewer than 5% of rare diseases have an FDA-approved treatment. Closing the gap takes more than science. @Adora Ndu, our Chief Regulatory Affairs Officer, shares more about what we hope to build in D.C., and why every minute counts. The door is open: bit.ly/4yU8b4O

Now published in @Cardio_Therapy: the first peer-reviewed, contemporary real-world comparative effectiveness study of TTR stabilizers in ATTR-CM, evaluating our near-complete stabilizer. Full results: bit.ly/4AcFJN4

Many muscular dystrophies look alike. Genetic testing is what tells them apart. Dr. Matthew Wicklund (@UTHealthSA) and Cyndy go Behind the Mystery on @lifetimetv discussing what it means to get a confirmed diagnosis and live with LGMD2I/R9. Sept 25 at 7:00 am EDT & PDT.

As part of #AFibAwarenessMonth, we’re raising awareness of AF in ATTR-CM. AF occurs in up to 70% of patients with ATTR-CM, and new-onset AF is associated with an increased risk of cardiovascular hospitalization, yet its impact on disease management is still not fully understood. Learn more: bit.ly/3SRr2xU

New exploratory PROPEL 3 data shared at @EuroSPE #ESPE2026 show favorable trends beyond growth for oral infigratinib, including in sleep apnea and ear infection measures over 52 weeks. We also shared longer-term results, highlighting that oral infigratinib demonstrated sustained improvements in growth and body proportionality in children treated for up to three years in the PROPEL program. Learn more: bit.ly/3VjAqv5

At 26, Adrienne went to the ER with severe high blood pressure and left with an ADPKD diagnosis. This #PKDAwarenessDay, she shares her story of uncertainty, strength and advocacy on the #OnRarePodcast. Listen here: bit.ly/3T8369N

Our van tour brings no-cost #genetictesting to #ADH1 families. The kit: a cheek swab and an envelope. Two to three weeks later, results arrive. Simple as that.

New publication in the @American_Heart's @JAHA_AHA: Joint assessment of sTTR and NT-proBNP may refine staging and prognosis in #ATTRCM, while the results of our near-complete TTR stabilizer were consistent across baseline biomarker profiles. Learn more: bit.ly/4iJ7Mxd

We’re thrilled to have a late-breaking oral presentation on oral infigratinib for achondroplasia that will be shared at the Annual @EuroSPE (#ESPE2026) Meeting 2026. Additionally, two posters and one eposter will be shared on the achondroplasia and hypochondroplasia programs, including longer-term data for oral infigratinib in achondroplasia. bit.ly/4gLlsVO

$BBIO management will join fireside chats at 4 healthcare investor conferences this Sept: @WellsFargo Healthcare Conf – Boston, 9/9, @Official_Cantor Global Healthcare Conf – NYC, 9/10, @MorganStanley Healthcare Conf – NYC, 9/1, & Bernstein Healthcare Leaders – NYC, 9/23. Learn more: bit.ly/4hMZMuS

New analyses in ATTR-CM were presented at @ESCcardio #ESC2026, including serial cardiac MRI data through Month 42 and open-label extension data through Month 54. Read the full release: bit.ly/46sS41O

Thank you, Ashley, for sharing your story with us. Your voice offers insight and hope to others living with #EPP and reminds us why these conversations matter. Listen on the #OnRarePodcast: bit.ly/4yWorTK

No clinic. No cost. Our mobile #genetictesting van is bringing answers directly to families across the West Coast – and once we find a person with ADH1, we can test the whole family. Follow the tour. #ADH1

First participant dosed in ASCEND-ATTR, a Phase 3b/4 study evaluating the long-term effects of acoramidis on cardiac structure, function & amyloid burden in ATTR-CM. This builds on our CMR substudy findings from ATTRibute-CM, which we will share updated data for at @ESCardio Congress 2026. Learn more: bit.ly/4wGNgk9 #ESC2026

We are thrilled to share two oral presentations and three posters on new ATTR-CM data at the @ESCardio Congress 2026. As part of BridgeBio's partnership with Yale's @cards_lab advance AI networks for earlier detection of ATTR-CM, three additional posters also will be presented. Learn more: bit.ly/4qut7fM

We're proud to announce our nomination for #PrixGalien's Best Product for Orphan Drug/Rare Diseases. This recognition reflects our commitment to addressing the unmet need for patients with ATTR-CM, a life-threatening progressive heart condition, and our broader mission to design transformative medicines for patients with genetic diseases. Congratulations to our fellow nominees and thank you to The Galien Foundation for this recognition. Read more: bit.ly/4ghKGeg

Join us on the 1st day of #ESC26 to hear experts discuss how timely detection matters in #ATTR-CM, emerging AI screening approaches and the clinical outcomes observed with early therapy initiation. Friday, Aug 28 | 10:00 AM CEST.

New episode of #OnRarePodcast: Pete Schmidt, MD, MSc., Chief Medical Officer of GondolaBio, joins Ashley, who lives with EPP, to explain the science behind EPP and XLP, from severe photosensitivity to the risk of liver damage. Listen: bit.ly/4yWorTK #OnRare #EPP #RareDisease

We reported our Q2 2026 financial results today. Full results here to take a look at what’s ahead: bit.ly/4bAKnKb

New episode of #OnRare: Ashley lived with undiagnosed EPP for over 40 years, including a liver crisis that led to a transplant. Hear her decades-long journey to diagnosis. Listen: bit.ly/4yWorTK #OnRare #EPP #RareDisease