| 1 |
Current and Emerging Clinical Treatment in Mitochondrial Disease |
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| Rory J. Tinker, Albert Z. Lim, Renae J. Stefanetti, Robert McFarland |
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| Molecular Diagnosis & Therapy. 2021; 25(2): 181 |
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| 2 |
Who funds Alzheimer's disease drug development? |
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| Jeffrey Cummings, Justin Bauzon, Garam Lee |
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| Alzheimer's & Dementia: Translational Research & Clinical Interventions. 2021; 7(1) |
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| 3 |
Economic evaluation of orphan drug Lutetium-Octreotate vs. Octreotide long-acting release for patients with an advanced midgut neuroendocrine tumour in the Netherlands |
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| Marije E. Hagendijk, Simon van der Schans, Cornelis Boersma, Maarten J. Postma, Simon van der Pol |
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| The European Journal of Health Economics. 2021; 22(6): 991 |
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| 4 |
An overview of cancer drugs approved through expedited approval programs and orphan medicine designation globally between 2011 and 2020 |
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| Shuhang Wang, Qiuyan Yang, Lan Deng, Qi Lei, Yuqi Yang, Peiwen Ma, Yuxin Men, Bryant C. Yung, Robert J. Lee, Mengzi Zhang, Ning Li |
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A Comparative Study of Pharmaceutical Incentives to Patents in India, USA and Europe |
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| Renu Kadian, Arun Nanda |
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| Applied Drug Research, Clinical Trials and Regulatory Affairs. 2021; 8(1): 12 |
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| 6 |
Access and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review With Content Analysis |
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| Adrienne Y.L. Chan, Vivien K.Y. Chan, Sten Olsson, Min Fan, Mark Jit, Mengchun Gong, Shuyang Zhang, Mengqin Ge, Swathi Pathadka, Claudia C.Y. Chung, Brian H.Y. Chung, Celine S.L. Chui, Esther W. Chan, Gloria H.Y. Wong, Terry Y. Lum, Ian C.K. Wong, Patrick Ip, Xue Li |
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Exploring different approaches to improve the success of drug discovery and development projects: a review |
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| Geoffrey Kabue Kiriiri, Peter Mbugua Njogu, Alex Njoroge Mwangi |
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| Future Journal of Pharmaceutical Sciences. 2020; 6(1) |
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Challenges in orphan drug development and regulatory policy in China |
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| Alice Cheng,Zhi Xie |
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| Orphanet Journal of Rare Diseases. 2017; 12(1) |
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| 9 |
Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL) |
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| Lieven Annemans,Ségolčne Aymé,Yann Le Cam,Karen Facey,Penilla Gunther,Elena Nicod,Michele Reni,Jean-Louis Roux,Michael Schlander,David Taylor,Carlo Tomino,Josep Torrent-Farnell,Sheela Upadhyaya,Adam Hutchings,Lugdivine Le Dez |
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| Orphanet Journal of Rare Diseases. 2017; 12(1) |
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| 10 |
Accelerated Access to Medicines |
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| Jessica Pace,Narcyz Ghinea,Ian Kerridge,Wendy Lipworth |
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| Therapeutic Innovation & Regulatory Science. 2017; 51(2): 157 |
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Cultural, geographical and ethical questions when looking to enroll pediatric patients in rare disease clinical trials |
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| Stefan Anzelewicz,Hanna Garnier,Arun Rangaswami,Piotr Czauderna |
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| Expert Opinion on Orphan Drugs. 2017; : 1 |
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Promoting innovation and access to quality, safe, efficacious, and affordable medicines for children: A China approach on the 69th
World Health Assembly |
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| Lingli Zhang,Bo Li,Zhanqiang Liu,Mengtao Li,Hong Zheng,Yang Sun |
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| Journal of Evidence-Based Medicine. 2017; 10(2): 70 |
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Using phase II data for the analysis of phase III studies: An application in rare diseases |
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| Simon Wandel,Beat Neuenschwander,Christian Röver,Tim Friede |
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| Clinical Trials. 2017; 14(3): 277 |
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| 14 |
New concepts on the therapeutic control of complement anaphylatoxin receptors |
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| Owen A. Hawksworth,Xaria X. Li,Liam G. Coulthard,Ernst J. Wolvetang,Trent M. Woodruff |
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“Good news is not always a positive market signal”—Investors activity around orphan drug designation |
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| Tiran Rothman |
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Design of a framework for the deployment of collaborative independent rare disease-centric registries: Gaucher disease registry model |
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| Matthew I. Bellgard,Kathryn R. Napier,Alan H. Bittles,Jeffrey Szer,Sue Fletcher,Nikolajs Zeps,Adam A. Hunter,Jack Goldblatt |
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A Single-use Strategy to Enable Manufacturing of Affordable Biologics |
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| Renaud Jacquemart,Melissa Vandersluis,Mochao Zhao,Karan Sukhija,Navneet Sidhu,Jim Stout |
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| Computational and Structural Biotechnology Journal. 2016; |
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A phase I study to repurpose disulfiram in combination with temozolomide to treat newly diagnosed glioblastoma after chemoradiotherapy |
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| Jiayi Huang,Jian L. Campian,Amit D. Gujar,David D. Tran,A. Craig Lockhart,Todd A. DeWees,Christina I. Tsien,Albert H. Kim |
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| 19 |
Factors influencing the difference between forecasted and actual drug sales volumes under the price–volume agreement in South Korea |
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| Sun-Young Park,Euna Han,Jini Kim,Eui-Kyung Lee |
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| Health Policy. 2016; |
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| 20 |
A review of the progress and pitfalls of FDA policy process: Planning a pathway for pharmaceutical interventions for hearing loss development |
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| Tanisha L. Hammill |
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| Hearing Research. 2016; |
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| 21 |
Pain in an era of armed conflicts: Prevention and treatment for warfighters and civilian casualties |
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| E. George,I. Elman,L. Becerra,Sheri Berg,D. Borsook |
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| 22 |
Static and Dynamic Properties of Fluorinated 4-Aryl-1,5-Benzodiazepinones |
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| Olga Martín,Marta Pérez-Torralba,M. Ángeles García,Rosa M. Claramunt,M. Carmen Torralba,M. Rosario Torres,Ibon Alkorta,José Elguero |
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| 23 |
Quantifying the persisting orphan-drug shortage public health crisis in the United States |
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| Szymon Jaroslawski,Chiraz Azaiez,Daria Korchagina,Mondher Toumi |
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| Journal of Market Access & Health Policy. 2016; : 1269473 |
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| 24 |
Factors That Affect the Acquisition of Reward Premiums for Promotion of Innovative Drug Discovery in Japan |
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| Shoyo Shibata,Ryotaro Uemura,Takeshi Suzuki |
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| Therapeutic Innovation & Regulatory Science. 2016; 50(1): 56 |
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| 25 |
Orphan Drugs and Potential Novel Approaches for Therapies of ß-Thalassemia: Current Status and Future Expectations |
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| Alessia Finotti,Monica Borgatti,Nicoletta Bianchi,Cristina Zuccato,Ilaria Lampronti,Roberto Gambari |
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| Expert Opinion on Orphan Drugs. 2016; 4(3): 299 |
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| 26 |
In Vitro Screening for Drug Repositioning |
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| Graeme F. Wilkinson,Kevin Pritchard |
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| Journal of Biomolecular Screening. 2015; 20(2): 167 |
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| 27 |
Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries |
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| Todd Gammie,Christine Y. Lu,Zaheer Ud-Din Babar,Silvio Garattini |
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| PLOS ONE. 2015; 10(10): e0140002 |
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| 28 |
Effectiveness, safety and costs of orphan drugs: an evidence-based review |
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| Igho J Onakpoya,Elizabeth A Spencer,Matthew J Thompson,Carl J Heneghan |
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| 29 |
Novel anti-glioblastoma agents and therapeutic combinations identified from a collection of FDA approved drugs |
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| Pengfei Jiang,Rajesh Mukthavavam,Ying Chao,Ila Bharati,Valentina Fogal,Sandra Pastorino,Xiuli Cong,Natsuko Nomura,Matt Gallagher,Taher Abbasi,Shireen Vali,Sandeep C Pingle,Milan Makale,Santosh Kesari |
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| Journal of Translational Medicine. 2014; 12(1): 13 |
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| 30 |
Estimating the budget impact of orphan drugs in Sweden and France 2013–2020 |
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| Adam Hutchings,Carina Schey,Richard Dutton,Felix Achana,Karolina Antonov |
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| Orphanet Journal of Rare Diseases. 2014; 9(1): 22 |
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| 31 |
The use of the United States FDA programs as a strategy to advance the development of drug products for neglected tropical diseases |
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| Kristina Sachs-Barrable,Jocelyn Conway,Pavel Gershkovich,Fady Ibrahim,Kishor M. Wasan |
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| Drug Development and Industrial Pharmacy. 2014; : 1 |
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| 32 |
A novel approach to sterile pharmaceutical freeze-drying |
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| Christopher Lee Albert Cherry,Huw Millward,Rose Cooper,John Landon |
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| Pharmaceutical Development and Technology. 2013; : 1 |
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| 33 |
Benefits of investment into modern medicines in Central–Eastern European countries |
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| András Inotai,Guenka Petrova,Dinko Vitezic,Zoltán Kaló |
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| Expert Review of Pharmacoeconomics & Outcomes Research. 2013; : 1 |
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| 34 |
Product lifecycle management through patents and regulatory strategies |
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| Vandana Prajapati,Swagat Tripathy,Harish Dureja |
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| Journal of Medical Marketing: Device, Diagnostic and Pharmaceutical Marketing. 2013; 13(3): 171 |
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Registering medicines for low-income countries: how suitable are the stringent review procedures of the World Health Organisation, the US Food and Drug Administration and the European Medicines Agency? |
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| Joachim Y. Doua,Jean-Pierre Van Geertruyden |
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When Everyone Is an Orphan: Against Adopting a U.S.-Styled Orphan Drug Policy in Canada |
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| Matthew Herder |
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| 37 |
Lipid Control in the Modern Era |
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| Richard V. Milani,Carl J. Lavie |
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| Journal of the American College of Cardiology. 2013; 62(23): 2185 |
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Orphan drugs: the regulatory environment |
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| Pedro Franco |
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| Drug Discovery Today. 2013; 18(3-4): 163 |
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Financing drug discovery for orphan diseases |
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| David E. Fagnan,Austin A. Gromatzky,Roger M. Stein,Jose-Maria Fernandez,Andrew W. Lo |
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Providing Medical Information for Orphan Drugs |
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| Sean Turbeville,David A. Wells,Carl S. Hornfeldt |
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| Drug Information Journal. 2012; 46(1): 88 |
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Optimizing efficacy of mucosal vaccines |
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| Ayman Gebril,Manal Alsaadi,Reinaldo Acevedo,Alexander B Mullen,Valerie A Ferro |
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| Expert Review of Vaccines. 2012; 11(9): 1139 |
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| 42 |
Orphan/rare drug discovery through drug repositioning |
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| Ramaiah Muthyala |
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| Drug Discovery Today: Therapeutic Strategies. 2011; 8(3-4): 71 |
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