Treatment of hereditary and acquired forms of transthyretin amyloidosis in the era of personalized medicine: the role of randomized controlled trials
Author:
Affiliation:
1. The Scripps Research Institute, San Diego, CA, USA;
2. The Scintillon Institute, San Diego, CA, USA;
3. Protego Biopharma, San Diego, CA, USA
Publisher
Informa UK Limited
Subject
Internal Medicine
Link
https://www.tandfonline.com/doi/pdf/10.1080/13506129.2019.1575201
Reference70 articles.
1. Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial
2. Repurposing Diflunisal for Familial Amyloid Polyneuropathy
3. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
4. Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
5. Personalized medicine: Time for one-person trials
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