Literature DB >> 12802033

Clinical trials and rare diseases.

Stephen W Lagakos.   

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Year:  2003        PMID: 12802033     DOI: 10.1056/NEJMe030024

Source DB:  PubMed          Journal:  N Engl J Med        ISSN: 0028-4793            Impact factor:   91.245


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  15 in total

1.  Development and use of new therapeutics for rare diseases: views from patients, caregivers, and advocates.

Authors:  Aaron S Kesselheim; Sarah McGraw; Lauren Thompson; Kelly O'Keefe; Joshua J Gagne
Journal:  Patient       Date:  2015-02       Impact factor: 3.883

2.  Policy alternatives for treatments for rare diseases.

Authors:  Abbas H Panju; Chaim M Bell
Journal:  CMAJ       Date:  2010-07-12       Impact factor: 8.262

3.  Outcome of systemic sclerosis associated interstitial lung disease treated with intravenous cyclophosphamide.

Authors:  Abhishek Abhishek; Ramin Yazdani; Fiona Pearce; Marian Regan; Ken Lim; Richard Hubbard; Peter Lanyon
Journal:  Clin Rheumatol       Date:  2011-04-12       Impact factor: 2.980

4.  A quantitative assessment of alkaptonuria: testing the reliability of two disease severity scoring systems.

Authors:  Trevor F Cox; Lakshminarayan Ranganath
Journal:  J Inherit Metab Dis       Date:  2011-07-09       Impact factor: 4.982

5.  Low-impact laparoscopic cholecystectomy is associated with decreased postoperative morbidity in patients with sickle cell disease.

Authors:  Nicola de'Angelis; Solafah Abdalla; Maria Clotilde Carra; Vincenzo Lizzi; Aleix Martínez-Pérez; Anoosha Habibi; Pablo Bartolucci; Frédéric Galactéros; Alexis Laurent; Francesco Brunetti
Journal:  Surg Endosc       Date:  2017-11-02       Impact factor: 4.584

Review 6.  Challenges in the design and conduct of therapeutic trials in channel disorders.

Authors:  Shannon L Venance; Barbara E Herr; Robert C Griggs
Journal:  Neurotherapeutics       Date:  2007-04       Impact factor: 7.620

7.  Epidemiological and Outcomes Research in Children with Pediatric Cardiomyopathy: Discussions from the International Workshop on Primary and Idiopathic Cardiomyopathies in Children.

Authors:  Steven E Lipshultz; James D Wilkinson
Journal:  Prog Pediatr Cardiol       Date:  2008-04

8.  Clinical research for rare disease: opportunities, challenges, and solutions.

Authors:  Robert C Griggs; Mark Batshaw; Mary Dunkle; Rashmi Gopal-Srivastava; Edward Kaye; Jeffrey Krischer; Tan Nguyen; Kathleen Paulus; Peter A Merkel
Journal:  Mol Genet Metab       Date:  2008-11-13       Impact factor: 4.797

Review 9.  How to Value Orphan Drugs? A Review of European Value Assessment Frameworks.

Authors:  Alessandra Blonda; Yvonne Denier; Isabelle Huys; Steven Simoens
Journal:  Front Pharmacol       Date:  2021-05-12       Impact factor: 5.810

10.  Initial Laparotomy Versus Peritoneal Drainage in Extremely Low Birthweight Infants With Surgical Necrotizing Enterocolitis or Isolated Intestinal Perforation: A Multicenter Randomized Clinical Trial.

Authors:  Martin L Blakely; Jon E Tyson; Kevin P Lally; Susan R Hintz; Barry Eggleston; David K Stevenson; Gail E Besner; Abhik Das; Robin K Ohls; William E Truog; Leif D Nelin; Brenda B Poindexter; Claudia Pedroza; Michele C Walsh; Barbara J Stoll; Rachel Geller; Kathleen A Kennedy; Reed A Dimmitt; Waldemar A Carlo; C Michael Cotten; Abbot R Laptook; Krisa P Van Meurs; Kara L Calkins; Gregory M Sokol; Pablo J Sanchez; Myra H Wyckoff; Ravi M Patel; Ivan D Frantz; Seetha Shankaran; Carl T D'Angio; Bradley A Yoder; Edward F Bell; Kristi L Watterberg; Colin A Martin; Carroll M Harmon; Henry Rice; Arlet G Kurkchubasche; Karl Sylvester; James C Y Dunn; Troy A Markel; Diana L Diesen; Amina M Bhatia; Alan Flake; Walter J Chwals; Rebeccah Brown; Kathryn D Bass; Shawn D St Peter; Christina M Shanti; Walter Pegoli; David Skarda; Joel Shilyansky; David G Lemon; Ricardo A Mosquera; Myriam Peralta-Carcelen; Ricki F Goldstein; Betty R Vohr; Isabell B Purdy; Abbey C Hines; Nathalie L Maitre; Roy J Heyne; Sara B DeMauro; Elisabeth C McGowan; Kimberly Yolton; Howard W Kilbride; Girija Natarajan; Kelley Yost; Sarah Winter; Tarah T Colaizy; Matthew M Laughon; Satyanarayana Lakshminrusimha; Rosemary D Higgins
Journal:  Ann Surg       Date:  2021-10-01       Impact factor: 13.787

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