A patient death in a CRISPR trial halts gene editing studies, raising critical safety questions. What this means for the ...

Since 2019, three disease-specific therapies have been approved to treat patients with ATTR-CM. The first was Pfizer’s Vyndamax (tafamidis) in May 2019, which was approved for both wild type and ...

NTLA shares plunge 55% in three months after the FDA placed a clinical hold on Nex-Z, suspending trials and milestone guidance following the death of a patient.

The global amyloidosis treatment market is on a trajectory of significant expansion, driven by heightened disease awareness, ...