ReviewDrug design, development and therapy2021
Drug Discovery and Development in Rare Diseases: Taking a Closer Look at the Tafamidis Story.
Review in Drug design, development and therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
27 citing papers in PubMed, 55 citations in OpenAlex.
- Long-term efficacy of tafamidis in patients with transthyretin amyloid cardiomyopathy by National Amyloidosis Centre stage.European journal of heart failure · 2025Trial
- On the Two-Step Hybrid Design for Augmenting Randomized Trials Using Real-World Data.Statistics in biopharmaceutical research · 2025Article
- Safety and Efficacy of Tafamidis in Chinese Patients with Transthyretin Amyloid Cardiomyopathy.Cardiology and therapy · 2025Article
- Transthyretin amyloid cardiomyopathy: Literature review and red-flag symptom clusters for each cardiology specialty.ESC heart failure · 2025Review
- Global epidemiology of heart failure.Nature reviews. Cardiology · 2024Review
- Safety assessment of Tafamidis: a real-world pharmacovigilance study of FDA adverse event reporting system (FAERS) events.BMC pharmacology & toxicology · 2024Article
- Adherence and persistence to tafamidis treatment among Medicare beneficiaries in the presence of a patient assistance program.Scientific reports · 2024Article
- Tafamidis therapy in transthyretin amyloid cardiomyopathy: a narrative review from clinical trials and real-world evidence.The Egyptian heart journal : (EHJ) : official bulletin of the Egyptian Society of Cardiology · 2024Review
- Real-World Effectiveness of High-Dose Tafamidis on Neurologic Disease Progression in Mixed-Phenotype Variant Transthyretin Amyloid Cardiomyopathy.Cardiology and therapy · 2024Article
- Development of Benziodarone Analogues with Enhanced Potency for Selective Binding to Transthyretin in Human Plasma.Journal of medicinal chemistry · 2024Article
- Tracing genetic diversity captures the molecular basis of misfolding disease.Nature communications · 2024Article
- A Comprehensive Review on Chemistry and Biology of Tafamidis in Transthyretin Amyloidosis.Mini reviews in medicinal chemistry · 2024Review
- The future of rare disease drug development: the rare disease cures accelerator data analytics platform (RDCA-DAP).Journal of pharmacokinetics and pharmacodynamics · 2023Review
- Variation of tafamidis plasma levels during the treatment of TTR amyloidosis patients with Glu89Gln mutation.European journal of clinical pharmacology · 2023Article
- Study methodology and insights from the palovarotene clinical development program in fibrodysplasia ossificans progressiva.BMC medical research methodology · 2023Article
- Binding of a Pyrene-Based Fluorescent Amyloid Ligand to Transthyretin: A Combined Crystallographic and Molecular Dynamics Study.The journal of physical chemistry. B · 2023Article
- De Novo Large Deletions in theDiagnostics (Basel, Switzerland) · 2023Article
- Trend of clinical trials of new drugs for rare diseases in China in recent 10 years.Orphanet journal of rare diseases · 2023Article
- Amyloid transthyretin cardiac amyloidosis with different manifestations, test findings and types.BMJ case reports · 2023Article
- Transthyretin Binding Mode Dichotomy of FluorescentACS chemical neuroscience · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Rare diseases are increasingly recognized as a global public health priority. Governments worldwide currently provide important incentives to stimulate the discovery and development of orphan drugs for the treatment of these conditions, but substantial scientific, clinical, and regulatory challenges remain. Tafamidis is a first-in-class, disease-modifying transthyretin (TTR) kinetic stabilizer that represents a major breakthrough in the treatment of transthyretin amyloidosis (ATTR amyloidosis). ATTR amyloidosis is a rare, progressive, and fatal systemic disorder caused by aggregation of misfolded TTR and extracellular deposition of amyloid fibrils in various tissues and organs, including the heart and nervous systems. In this review, we present the successful development of tafamidis spanning 3 decades, marked by meticulous laboratory research into disease mechanisms and natural history, and innovative clinical study design and implementation. These efforts established the safety and efficacy profile of tafamidis, leading to its regulatory approval, and enabled post-approval initiatives that further support patients with ATTR amyloidosis.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.