News

“We hope you will join us for SLC6A1 Connect’s 2024 International Scientific Symposium and Family Conference to be held on December 5-6, 2024 at the Omni Hotel in…

Join the LGS Community on December 5th before AES for a special showing of It’s All Your Fault, Tyler Price! – a new musical from Emmy winner Ben Decter (Lucifer)…

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…