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The purpose of this symposium is to bring established investigators, junior researchers and industrials together to discuss and expand this exciting area of…

For people living with rare diseases, finding financial help for out-of-pocket cost is vital—the PAN Foundation can help. Join to learn how you can apply for a PAN…

PITTSBURGH, Nov. 26, 2024 /PRNewswire/ -- PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, is pleased…

On a Zoom call last month from California, BridgeBio CEO Neil Kumar needled a colleague about the remote possibility of the New York Yankees rallying to win the…

Acoramidis (Attruby) gained FDA approval for treating wild-type or variant transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM), BridgeBio Pharma announced.…

BridgeBio secured approval for Attruby Friday, along with the all-important mortality benefit that could give the drug a significant boost in the market against…

Transthyretin amyloid cardiomyopathy (ATTR-CM) patients treated with the amyloidosis drug acoramidis are associated with significantly lower mortality and…

Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. Attruby has been shown to preserve the native function of…

- Acoramidis demonstrated the earliest known time to separation in cardiovascular outcomes in the ATTRibute-CM study (3 months), with statistically significant risk…

Net cash used in operating and investing activities was $45.2 million and $102.5 million for the third quarter and first nine months of 2024, respectively; quarter-…