Developing Therapy for Transthyretin Amyloidosis - 07/05/22
, Kathleen Zhang, MD a, Daniel J. Lenihan, MD a, Ronald Witteles, MD bAbstract |
Transthyretin amyloidosis (ATTR) is an under-recognized cause of cardiomyopathy and neuropathy. Until recently, there were limited therapeutic options for ATTR. However, new therapeutics, including tafamidis, patisiran, and inotersen, increase both quality and length of life in patients with ATTR. This review details the chronological development of ATTR therapies through landmark clinical trials. In addition, we discuss emerging ATTR therapies including improvements in drug delivery methods, antibodies to break down deposited amyloid fibrils, and gene editing. ATTR is a prime example of how an understanding of the pathophysiological basis of disease can lead to effective therapies. The future of ATTR therapy is bright, with every reason to believe outcomes will continue to improve.
Le texte complet de cet article est disponible en PDF.Keywords : ATTR, Cardiomyopathy, Inotersen, Neuropathy, Patisiran, Tafamidis
Plan
| Funding: None. |
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| Conflicts of Interest: CMC reports research support from Alnylam Pharmaceuticals and Pfizer Inc.; and consulting fees from Alnylam. KZ reports consulting fees from Eidos Therapeutics. DJL reports research support from Alnylam, Eidos, Prothena Corporation, Pfizer Inc., and Jazz Pharmaceuticals; and consulting fees from Bristol-Myers Squibb, Ipsen Biopharmaceuticals, Prothena, Acorda Therapeutics, Lilly (Eli) USA, and Roche Pharmaceutical. RW reports consulting fees from Pfizer Inc., Alnylam, Eidos, Regeneron, and Ionis/Akcea. |
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| Authorship: All authors had access to the data and a role in writing the manuscript. |
Vol 135 - N° S1
P. S44-S48 - avril 2022 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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