PALO ALTO, Calif., March 31, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today ...
Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, often fatal disease. Nexiguran ziclumeran (nex-z) is an investigational therapy based on CRISPR-Cas9 (clustered regularly ...
Alnylam’s closely watched launch of its rare disease drug Amvuttra continued to outpace Wall Street expectations. Riding its March expansion into the heart condition of transthyretin amyloid ...
The US Food and Drug Administration (FDA) has declined to approve the RNA interference (RNAi) therapeutic agent patisiran (Onpattro, Alnylam Pharmaceuticals) for treatment of transthyretin-mediated ...
The RNA interference (RNAi) therapeutic patisiran (Onpattro, Alnylam Pharmaceuticals) led to statistically significant improvement in functional capacity and quality of life in adults with ...
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