标题
Drug Repurposing for Rare Diseases
作者
关键词
drug discovery, drug repositioning, drug repurposing, genetic diseases, rare diseases
出版物
TRENDS IN PHARMACOLOGICAL SCIENCES
Volume 42, Issue 4, Pages 255-267
出版商
Elsevier BV
发表日期
2021-02-07
DOI
10.1016/j.tips.2021.01.003
参考文献
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注意:仅列出部分参考文献,下载原文获取全部文献信息。- On-Label or Off-Label? Overcoming Regulatory and Financial Barriers to Bring Repurposed Medicines to Cancer Patients
- (2020) Ciska Verbaanderd et al. Frontiers in Pharmacology
- Genetic Modifiers and Rare Mendelian Disease
- (2020) K. M. Tahsin Hassan Rahit et al. Genes
- Treatment of Fabry's Disease With Migalastat: Outcome From a Prospective Observational Multicenter Study (FAMOUS)
- (2020) Malte Lenders et al. CLINICAL PHARMACOLOGY & THERAPEUTICS
- Exogenous Flupirtine as Potential Treatment for CLN3 Disease
- (2020) Katia Maalouf et al. Cells
- Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis
- (2019) Scott D. Solomon et al. CIRCULATION
- Induced pluripotent stem cells for neural drug discovery
- (2019) Atena Farkhondeh et al. DRUG DISCOVERY TODAY
- A Computational Framework for Genome-wide Characterization of the Human Disease Landscape
- (2019) Young-suk Lee et al. Cell Systems
- The patient’s view on rare disease trial design – a qualitative study
- (2019) C. M. W. Gaasterland et al. Orphanet Journal of Rare Diseases
- Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
- (2019) Eliane Sardh et al. NEW ENGLAND JOURNAL OF MEDICINE
- From gene to treatment: supporting rare disease translational research through model systems
- (2019) Julija Hmeljak et al. Disease Models & Mechanisms
- A Diagnosis for All Rare Genetic Diseases: The Horizon and the Next Frontiers
- (2019) Kym M. Boycott et al. CELL
- Exploiting machine learning for end-to-end drug discovery and development
- (2019) Sean Ekins et al. NATURE MATERIALS
- Mutation-specific therapies and drug repositioning in cystic fibrosis
- (2019) Valeria R. Villella et al. MINERVA PEDIATRICA
- Genome sequencing and implications for rare disorders
- (2019) Jennifer E. Posey Orphanet Journal of Rare Diseases
- Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database
- (2019) Stéphanie Nguengang Wakap et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- The use or generation of biomedical data and existing medicines to discover and establish new treatments for patients with rare diseases – recommendations of the IRDiRC Data Mining and Repurposing Task Force
- (2019) Noel T Southall et al. Orphanet Journal of Rare Diseases
- Artificial Intelligence (AI) in Rare Diseases: Is the Future Brighter?
- (2019) Sandra Brasil et al. Genes
- Cantú syndrome: Findings from 74 patients in the International Cantú Syndrome Registry
- (2019) Dorothy K. Grange et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART C-SEMINARS IN MEDICAL GENETICS
- Cystic fibrosis transmembrane conductance regulator modulators
- (2018) Elizabeth B. Burgener et al. CURRENT OPINION IN PEDIATRICS
- Finding hidden treasures in old drugs: the challenges and importance of licensing generics
- (2018) Melek Simsek et al. DRUG DISCOVERY TODAY
- Association of Lonafarnib Treatment vs No Treatment With Mortality Rate in Patients With Hutchinson-Gilford Progeria Syndrome
- (2018) Leslie B. Gordon et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- Overcoming the legal and regulatory barriers to drug repurposing
- (2018) Alasdair Breckenridge et al. NATURE REVIEWS DRUG DISCOVERY
- Integrative omics for health and disease
- (2018) Konrad J. Karczewski et al. NATURE REVIEWS GENETICS
- Pluripotent Stem Cell Platforms for Drug Discovery
- (2018) Kevin G. Chen et al. TRENDS IN MOLECULAR MEDICINE
- Cardiac Abnormalities in Patients With Hutchinson-Gilford Progeria Syndrome
- (2018) Ashwin Prakash et al. JAMA Cardiology
- Advancing precision medicine with personalized drug screening
- (2018) Kirill Gorshkov et al. DRUG DISCOVERY TODAY
- Drug repurposing: progress, challenges and recommendations
- (2018) Sudeep Pushpakom et al. NATURE REVIEWS DRUG DISCOVERY
- From scientific discovery to treatments for rare diseases – the view from the National Center for Advancing Translational Sciences – Office of Rare Diseases Research
- (2018) Petra Kaufmann et al. Orphanet Journal of Rare Diseases
- Discovery of Aromatic Carbamates that Confer Neuroprotective Activity by Enhancing Autophagy and Inducing the Anti-Apoptotic Protein B-Cell Lymphoma 2 (Bcl-2)
- (2017) Nihar Kinarivala et al. JOURNAL OF MEDICINAL CHEMISTRY
- Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
- (2017) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Health-related quality of life among adults with diverse rare disorders
- (2017) Kathleen R. Bogart et al. Orphanet Journal of Rare Diseases
- Rare Diseases: Drug Discovery and Informatics Resource
- (2017) Mingzhu Zhao et al. Interdisciplinary Sciences-Computational Life Sciences
- Diagnostic needs for rare diseases and shared prediagnostic phenomena: Results of a German-wide expert Delphi survey
- (2017) Susanne Blöß et al. PLoS One
- ksRepo: a generalized platform for computational drug repositioning
- (2016) Adam S. Brown et al. BMC BIOINFORMATICS
- Drug repositioning approaches to parasitic diseases: a medicinal chemistry perspective
- (2016) Leonardo G. Ferreira et al. DRUG DISCOVERY TODAY
- Drug combination therapy increases successful drug repositioning
- (2016) Wei Sun et al. DRUG DISCOVERY TODAY
- Innovation in the pharmaceutical industry: New estimates of R&D costs
- (2016) Joseph A. DiMasi et al. JOURNAL OF HEALTH ECONOMICS
- Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
- (2016) Maria Sessa et al. LANCET
- Can you teach old drugs new tricks?
- (2016) Nicola Nosengo NATURE
- Induced pluripotent stem cell technology: a decade of progress
- (2016) Yanhong Shi et al. NATURE REVIEWS DRUG DISCOVERY
- The Prevalence of Huntington's Disease
- (2016) Michael D. Rawlins et al. NEUROEPIDEMIOLOGY
- Survey of healthcare experiences of Australian adults living with rare diseases
- (2016) Caron Molster et al. Orphanet Journal of Rare Diseases
- A survey of current trends in computational drug repositioning
- (2015) Jiao Li et al. BRIEFINGS IN BIOINFORMATICS
- Computational and Experimental Advances in Drug Repositioning for Accelerated Therapeutic Stratification
- (2015) Khader Shameer et al. CURRENT TOPICS IN MEDICINAL CHEMISTRY
- Drug repositioning can accelerate discovery of pharmacological chaperones
- (2015) Bruno Hay Mele et al. Orphanet Journal of Rare Diseases
- Impact of Farnesylation Inhibitors on Survival in Hutchinson-Gilford Progeria Syndrome
- (2014) Leslie B. Gordon et al. CIRCULATION
- Clinical Exome Sequencing for Genetic Identification of Rare Mendelian Disorders
- (2014) Hane Lee et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- Validating drug repurposing signals using electronic health records: a case study of metformin associated with reduced cancer mortality
- (2014) H. Xu et al. JOURNAL OF THE AMERICAN MEDICAL INFORMATICS ASSOCIATION
- Big data: the next frontier for innovation in therapeutics and healthcare
- (2014) Naiem T Issa et al. Expert Review of Clinical Pharmacology
- Clinical Trials in Rare Disease
- (2013) Erika F. Augustine et al. JOURNAL OF CHILD NEUROLOGY
- Epigenetic and genetic alterations of the imprinting disorder Beckwith–Wiedemann syndrome and related disorders
- (2013) Hidenobu Soejima et al. JOURNAL OF HUMAN GENETICS
- The promise of whole-exome sequencing in medical genetics
- (2013) Bahareh Rabbani et al. JOURNAL OF HUMAN GENETICS
- Rare-disease genetics in the era of next-generation sequencing: discovery to translation
- (2013) Kym M. Boycott et al. NATURE REVIEWS GENETICS
- Clinical Whole-Exome Sequencing for the Diagnosis of Mendelian Disorders
- (2013) Yaping Yang et al. NEW ENGLAND JOURNAL OF MEDICINE
- Treatment of Muckle-Wells syndrome: analysis of two IL-1-blocking regimens
- (2013) Jasmin B Kuemmerle-Deschner et al. ARTHRITIS RESEARCH & THERAPY
- The Emerging Paradigm of Network Medicine in the Study of Human Disease
- (2012) Stephen Y. Chan et al. CIRCULATION RESEARCH
- Rare Diseases: The Bane of Modern Society and the Quest for Cures
- (2012) N Azie et al. CLINICAL PHARMACOLOGY & THERAPEUTICS
- Network medicine: linking disorders
- (2012) Rosario M. Piro HUMAN GENETICS
- Clinical application of exome sequencing in undiagnosed genetic conditions
- (2012) Anna C Need et al. JOURNAL OF MEDICAL GENETICS
- Challenges in Whole Exome Sequencing: An Example from Hereditary Deafness
- (2012) Asli Sirmaci et al. PLoS One
- Clinical trial of a farnesyltransferase inhibitor in children with Hutchinson-Gilford progeria syndrome
- (2012) L. B. Gordon et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Human Genome Sequencing in Health and Disease
- (2011) Claudia Gonzaga-Jauregui et al. Annual Review of Medicine
- Drug repositioning for orphan diseases
- (2011) D. Sardana et al. BRIEFINGS IN BIOINFORMATICS
- In silico repositioning of approved drugs for rare and neglected diseases
- (2011) Sean Ekins et al. DRUG DISCOVERY TODAY
- Principles of early drug discovery
- (2010) JP Hughes et al. BRITISH JOURNAL OF PHARMACOLOGY
- Saturation of the Human Phenome
- (2010) Mark E. Samuels CURRENT GENOMICS
- Rational approaches to targeted polypharmacology: creating and navigating protein–ligand interaction networks
- (2010) James T Metz et al. CURRENT OPINION IN CHEMICAL BIOLOGY
- Mechanisms of Drug Toxicity and Relevance to Pharmaceutical Development
- (2010) F. Peter Guengerich Drug Metabolism and Pharmacokinetics
- Accelerating orphan drug development
- (2010) Timothy R. Coté et al. NATURE REVIEWS DRUG DISCOVERY
- Unveiling the role of network and systems biology in drug discovery
- (2010) Albert Pujol et al. TRENDS IN PHARMACOLOGICAL SCIENCES
- Pivotal studies of orphan drugs approved for neurological diseases
- (2009) Jun Mitsumoto et al. ANNALS OF NEUROLOGY
- Use of Canakinumab in the Cryopyrin-Associated Periodic Syndrome
- (2009) Helen J. Lachmann et al. NEW ENGLAND JOURNAL OF MEDICINE
- Genetic diagnosis by whole exome capture and massively parallel DNA sequencing
- (2009) Murim Choi et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Repurposing with a Difference
- (2009) M. S. Boguski et al. SCIENCE
- Drug development for orphan diseases in the context of personalized medicine
- (2009) George J. Brewer Translational Research
- Empowerment of patients: lessons from the rare diseases community
- (2008) Ségolène Aymé et al. LANCET
- Clinical research for rare disease: Opportunities, challenges, and solutions
- (2008) Robert C. Griggs et al. MOLECULAR GENETICS AND METABOLISM
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