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Literature DB >> 29685384

Registry Contributions to Strengthen Cell and Gene Therapeutic Evidence.

Mohamed Abou-El-Enein¹, David W Grainger², Sven Kili³.

Abstract

Mesh：

Year: 2018 PMID： 29685384 PMCID： PMC5993957 DOI： 10.1016/j.ymthe.2018.04.007

Source DB: PubMed Journal: Mol Ther ISSN： 1525-0016 Impact factor: 11.454

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10 in total

Review 1. Enhancing patient-level clinical data access to promote evidence-based practice and incentivize therapeutic innovation.

Authors: Alice Fortunato; David W Grainger; Mohamed Abou-El-Enein
Journal: Adv Drug Deliv Rev Date: 2018-01-31 Impact factor: 15.470

2. Challenges of rare disease research: limited patients and competing priorities.

Authors: M V Ragni; C G Moore; V Bias; N S Key; P A Kouides; C W Francis
Journal: Haemophilia Date: 2011-09-13 Impact factor: 4.287

3. The business case for cell and gene therapies.

Authors: Mohamed Abou-El-Enein; Gerhard Bauer; Petra Reinke
Journal: Nat Biotechnol Date: 2014-12 Impact factor: 54.908

4. Deciphering the EU clinical trials regulation.

Authors: Mohamed Abou-El-Enein; Christian K Schneider
Journal: Nat Biotechnol Date: 2016-03 Impact factor: 54.908

5. Overcoming Challenges Facing Advanced Therapies in the EU Market.

Authors: Mohamed Abou-El-Enein; Ahmed Elsanhoury; Petra Reinke
Journal: Cell Stem Cell Date: 2016-09-01 Impact factor: 24.633

6. The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal.

Authors: Robert Hettle; Mark Corbett; Sebastian Hinde; Robert Hodgson; Julie Jones-Diette; Nerys Woolacott; Stephen Palmer
Journal: Health Technol Assess Date: 2017-02 Impact factor: 4.014

Review 7. RD-Connect: an integrated platform connecting databases, registries, biobanks and clinical bioinformatics for rare disease research.

Authors: Rachel Thompson; Louise Johnston; Domenica Taruscio; Lucia Monaco; Christophe Béroud; Ivo G Gut; Mats G Hansson; Peter-Bram A 't Hoen; George P Patrinos; Hugh Dawkins; Monica Ensini; Kurt Zatloukal; David Koubi; Emma Heslop; Justin E Paschall; Manuel Posada; Peter N Robinson; Kate Bushby; Hanns Lochmüller
Journal: J Gen Intern Med Date: 2014-08 Impact factor: 5.128

8. Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making.

Authors: Marc L Berger; Harold Sox; Richard J Willke; Diana L Brixner; Hans-Georg Eichler; Wim Goettsch; David Madigan; Amr Makady; Sebastian Schneeweiss; Rosanna Tarricone; Shirley V Wang; John Watkins; C Daniel Mullins
Journal: Pharmacoepidemiol Drug Saf Date: 2017-09 Impact factor: 2.890

9. Innovative regenerative medicines in the EU: a better future in evidence?

Authors: Mark S Corbett; Andrew Webster; Robert Hawkins; Nerys Woolacott
Journal: BMC Med Date: 2017-03-08 Impact factor: 8.775

10. Registries supporting new drug applications.

Authors: Carla J Jonker; H Marijke van den Berg; Marcel S G Kwa; Arno W Hoes; Peter G M Mol
Journal: Pharmacoepidemiol Drug Saf Date: 2017-10-06 Impact factor: 2.890

10 in total

13 in total

Review 1. Paving the way for small-molecule drug discovery.

Authors: Yu-Shui Ma; Rui Xin; Xiao-Li Yang; Yi Shi; Dan-Dan Zhang; Hui-Min Wang; Pei-Yao Wang; Ji-Bin Liu; Kai-Jian Chu; Da Fu
Journal: Am J Transl Res Date: 2021-03-15 Impact factor: 4.060

Review 2. Concise Review: A Comprehensive Analysis of Reported Adverse Events in Patients Receiving Unproven Stem Cell-Based Interventions.

Authors: Gerhard Bauer; Magdi Elsallab; Mohamed Abou-El-Enein
Journal: Stem Cells Transl Med Date: 2018-07-31 Impact factor: 6.940

3. Systematic analysis using a bioinformatics strategy identifies SFTA1P and LINC00519 as potential prognostic biomarkers for lung squamous cell carcinoma.

Authors: Yu-Zhen Yin; Shi-Hua Yao; Chun-Guang Li; Yu-Shui Ma; Zhou-Jun Kang; Jia-Jia Zhang; Cheng-You Jia; Li-Kun Hou; Shan-Shan Qin; Xin Fan; Han Zhang; Meng-Die Yang; Dan-Dan Zhang; Gai-Xia Lu; Hui-Min Wang; Li-Peng Gu; Lin-Lin Tian; Pei-Yao Wang; Ping-Sheng Cao; Wei Wu; Zi-Yang Cao; Zhong-Wei Lv; Bo-Wen Shi; Chun-Yan Wu; Geng-Xi Jiang; Da Fu; Fei Yu
Journal: Am J Transl Res Date: 2021-01-15 Impact factor: 4.060

Review 4. Basic approaches, challenges and opportunities for the discovery of small molecule anti-tumor drugs.

Authors: Yu-Shui Ma; Ji-Bin Liu; Xiao-Li Yang; Rui Xin; Yi Shi; Dan-Dan Zhang; Hui-Min Wang; Pei-Yao Wang; Qin-Lu Lin; Wen Li; Da Fu
Journal: Am J Cancer Res Date: 2021-06-15 Impact factor: 6.166

5. Cell and Gene Therapy Trials: Are We Facing an 'Evidence Crisis'?

Authors: Mohamed Abou-El-Enein; Spencer Phillips Hey
Journal: EClinicalMedicine Date: 2019-02-02

Review 6. Reflection on the enactment and impact of safety laws for regenerative medicine in Japan.

Authors: Kayo Takashima; Michael Morrison; Jusaku Minari
Journal: Stem Cell Reports Date: 2021-05-20 Impact factor: 7.765

7. Evidence generation and reproducibility in cell and gene therapy research: A call to action.

Authors: Mohamed Abou-El-Enein; Aris Angelis; Frederick R Appelbaum; Nancy C Andrews; Susan E Bates; Arlene S Bierman; Malcolm K Brenner; Marina Cavazzana; Michael A Caligiuri; Hans Clevers; Emer Cooke; George Q Daley; Victor J Dzau; Lee M Ellis; Harvey V Fineberg; Lawrence S B Goldstein; Stephen Gottschalk; Margaret A Hamburg; Donald E Ingber; Donald B Kohn; Adrian R Krainer; Marcela V Maus; Peter Marks; Christine L Mummery; Roderic I Pettigrew; Joni L Rutter; Sarah A Teichmann; Andre Terzic; Fyodor D Urnov; David A Williams; Jedd D Wolchok; Mark Lawler; Cameron J Turtle; Gerhard Bauer; John P A Ioannidis
Journal: Mol Ther Methods Clin Dev Date: 2021-07-21 Impact factor: 6.698