Literature DB >> 26584589

Challenges of clinical trial design for DMD.

Valeria Ricotti1, Francesco Muntoni2, Thomas Voit3.   

Abstract

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Year:  2015        PMID： 26584589     DOI： 10.1016/j.nmd.2015.10.007

Source DB:  PubMed          Journal:  Neuromuscul Disord        ISSN： 0960-8966            Impact factor:   4.296


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  7 in total

Review 1.  Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy.

Authors:  Luca Bello; Elena Pegoraro
Journal:  Acta Myol       Date:  2016-12

2.  Dystrophin's central domain forms a complex filament that becomes disorganized by in-frame deletions.

Authors:  Olivier Delalande; Anne-Elisabeth Molza; Raphael Dos Santos Morais; Angélique Chéron; Émeline Pollet; Céline Raguenes-Nicol; Christophe Tascon; Emmanuel Giudice; Marine Guilbaud; Aurélie Nicolas; Arnaud Bondon; France Leturcq; Nicolas Férey; Marc Baaden; Javier Perez; Pierre Roblin; France Piétri-Rouxel; Jean-François Hubert; Mirjam Czjzek; Elisabeth Le Rumeur
Journal:  J Biol Chem       Date:  2018-03-13       Impact factor: 5.157

Review 3.  Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy.

Authors:  Giulia Ricci; Luca Bello; Francesca Torri; Erika Schirinzi; Elena Pegoraro; Gabriele Siciliano
Journal:  Neurol Sci       Date:  2022-05-24       Impact factor: 3.830

4.  The Action Duchenne 13th International Annual Conference, November 6-7, 2015, London, UK.

Authors:  Diana Ribeiro; Daniel W Stuckey
Journal:  EBioMedicine       Date:  2015-12-03       Impact factor: 8.143

Review 5.  Splice-switching antisense oligonucleotides as therapeutic drugs.

Authors:  Mallory A Havens; Michelle L Hastings
Journal:  Nucleic Acids Res       Date:  2016-06-10       Impact factor: 16.971

Review 6.  Eteplirsen in the treatment of Duchenne muscular dystrophy.

Authors:  Kenji Rowel Q Lim; Rika Maruyama; Toshifumi Yokota
Journal:  Drug Des Devel Ther       Date:  2017-02-28       Impact factor: 4.162

7.  Development of a model-based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy.

Authors:  Karthik Lingineni; Varun Aggarwal; Juan Francisco Morales; Daniela J Conrado; Diane Corey; Camille Vong; Jackson Burton; Jane Larkindale; Klaus Romero; Stephan Schmidt; Sarah Kim
Journal:  CPT Pharmacometrics Syst Pharmacol       Date:  2022-01-03
  7 in total

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