BridgeBio

We would like to extend our deepest sympathies to the friends, family, and colleagues of Clinical Professor Dr. Jignesh Patel, who recently passed away. His remarkable contributions to the field of cardiology have left an indelible mark on medical research and patient care. Dr. Patel was a true trailblazer, and his legacy will continue to inspire future generations of healthcare professionals. Our thoughts are with all who were touched by his work and his life.

Our CEO Neil Kumar joined Liz Claman on Fox Business Network’s Claman Countdown to discuss BridgeBio’s mission to develop groundbreaking medicines while ensuring affordability for patients and highlighted what’s ahead to bring transformative therapies to those in need. View segment here: https://bit.ly/418QCyz

In our latest #OnRarePodcast episode, Kady shares the emotional journey of raising her 4-year-old son, Julien, who is living with a unique case of autosomal dominant hypocalcemia type 1 (ADH1). Managing his condition has been a daily balancing act, as Julien’s specific mutation is so rare that he is the only known case in the world. Listen to their story here: https://bit.ly/41adKwz 

Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD 2I/R9) is a rare genetic disorder that causes progressive muscle weakness around the hips and shoulders. However, LGMD 2I/R9 can also impact the heart. This is one reason why getting genetic confirmation of your LGMD subtype is important.  Some individuals with LGMD 2I/R9 may develop cardiomyopathy, weakening of the heart muscles and conduction abnormalities (irregular heartbeats). Obtaining confirmation of your subtype will allow physicians to monitor your heart and provide optimal care.  💙 Knowledge is power—stay proactive about your heart health. #LGMD #GeneticTesting #CardiacHealth #MuscularDystrophy #DiseaseAwareness 

𝗕𝗲𝗵𝗶𝗻𝗱 𝘁𝗵𝗲 𝗦𝗰𝗲𝗻𝗲𝘀: 𝗧𝗵𝗲 𝗦𝗰𝗶𝗲𝗻𝗰𝗲 𝗼𝗳 𝗗𝗿𝘂𝗴 𝗗𝗶𝘀𝗰𝗼𝘃𝗲𝗿𝘆 Transthyretin amyloid cardiomyopathy (ATTR-CM) is a complex heart condition caused by amyloid plaques from destabilized TTR. In collaboration with BridgeBio, researchers explored indirect exposure-response modeling to understand how Acoramidis improves patient outcomes by stabilizing TTR. The 𝗔𝗧𝗧𝗥𝗶𝗯𝘂𝘁𝗲-𝗖𝗠 𝗣𝗵𝗮𝘀𝗲 𝟯 𝘀𝘁𝘂𝗱𝘆 showed that 𝗔𝗰𝗼𝗿𝗮𝗺𝗶𝗱𝗶𝘀 helped reduce deaths by 25% compared to a placebo by 30 months. When they looked at how much transthyretin (TTR) levels changed after 28 days instead of direct exposure measures, they saw a strong connection with patient outcomes—patients with bigger increases in TTR had better survival rates and improved physical function. Adding factors like kidney function (eGFR) and baseline TTR levels gave an even clearer understanding of how well Acoramidis works. 𝗩𝗶𝗲𝘄 𝘁𝗵𝗲 𝘀𝗰𝗶𝗲𝗻𝗰𝗲 𝗵𝗲𝗿𝗲: https://lnkd.in/eUDf6eBz 𝗖𝗼𝗻𝘁𝗿𝗶𝗯𝘂𝘁𝗼𝗿𝘀: Satish Rao, Jean-François Tamby, MD-MBA, Uma Sinha, Surendhar Reddy Chepyala, PhD Reddy Chepyala, Brian Adam, Bill Poland 𝗟𝗲𝘁’𝘀 𝗖𝗼𝗻𝗻𝗲𝗰𝘁: Scan the QR code or visit www.certara.com to explore how exposure-response modeling can optimize drug development and regulatory decision-making. #DrugDevelopment #ExposureResponseModeling #Transthyretin #ATTRCM #Pharmacokinetics #ClinicalResearch #Certara

Meet Matthew R. Hawes, our next Rare Hero and a passionate advocate for the ATTR-CM and rare disease communities. From leading teams to raising awareness, Matt’s dedication stems from his deeply personal connection to the work. Read his full story here: https://lnkd.in/gJSxEUMD #rarediseaseday #rarehero #ATTRCM #patientvoices #rareadvocacy

#HFWeek2025 starts NOW! Here's how you can get involved 👇 ❤️ Tell your patients! ❤️ Share your #MyHFStory2025 ❤️ Highlight critical HF Stats ❤️ Empower patients, caregivers, and providers to take informed actions Learn more: https://lnkd.in/gTU2n6xH

Alongside our partners at Bayer | Pharmaceuticals, we look forward to this opportunity to serve ATTR-CM patients across the European Union.

We're pleased to share that the European Commission has approved our orally-administered near-complete (≥90%) stabilizer of TTR for the treatment of adult patients with transthyretin cardiac amyloidosis (ATTR-CM) in the EU. ATTR-CM is a progressive fatal disease that presents as an infiltrative, restrictive cardiomyopathy resulting in heart failure, and we are proud to partner with Bayer | Pharmaceuticals for all commercial activity for this treatment in the EU.  Read more here: https://bit.ly/417Xq0O

Aspa Therapeutics, BridgeBio’s Gene Therapy program, recently published findings correlating urine NAA levels with a clinical phenotype in #CanavanDisease. This study, using insights gained from our CVN-101 natural history study, demonstrates that in untreated children with Canavan disease, a milder phenotype is associated with lower levels NAA in the urine. In contrast, those with the more common and severe form of the disease show significantly higher NAA levels in the urine.  Read more from the publication now available online: https://bit.ly/40K5tk4