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Literature DB >> 25101726

EMA reconsiders 'read-through' drug against Duchenne muscular dystrophy following appeal.

Asher Mullard.

Abstract

Entities: Disease

Mesh：

Substances：
Oxadiazoles
ataluren

Year: 2014 PMID： 25101726 DOI： 10.1038/nbt0814-706

Source DB: PubMed Journal: Nat Biotechnol ISSN： 1087-0156 Impact factor: 54.908

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2 in total

1. Doubts raised over 'read-through' Duchenne drug mechanism.

Authors: Cormac Sheridan
Journal: Nat Biotechnol Date: 2013-09 Impact factor: 54.908

2. Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

Authors: Eitan Kerem; Michael W Konstan; Kris De Boeck; Frank J Accurso; Isabelle Sermet-Gaudelus; Michael Wilschanski; J Stuart Elborn; Paola Melotti; Inez Bronsveld; Isabelle Fajac; Anne Malfroot; Daniel B Rosenbluth; Patricia A Walker; Susanna A McColley; Christiane Knoop; Serena Quattrucci; Ernst Rietschel; Pamela L Zeitlin; Jay Barth; Gary L Elfring; Ellen M Welch; Arthur Branstrom; Robert J Spiegel; Stuart W Peltz; Temitayo Ajayi; Steven M Rowe
Journal: Lancet Respir Med Date: 2014-05-15 Impact factor: 30.700

2 in total

8 in total

Review 1. Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials.

Authors: Yuko Shimizu-Motohashi; Shouta Miyatake; Hirofumi Komaki; Shin'ichi Takeda; Yoshitsugu Aoki
Journal: Am J Transl Res Date: 2016-06-15 Impact factor: 4.060

EMA reconsiders 'read-through' drug against Duchenne muscular dystrophy following appeal.

1. Doubts raised over 'read-through' Duchenne drug mechanism.

2. Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

Review 1. Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials.

2. Characterization of new-generation aminoglycoside promoting premature termination codon readthrough in cancer cells.

3. Duchenne muscular dystrophy drugs at the crossroads, as newer agents advance.

4. Magnetic resonance imaging in facioscapulohumeral muscular dystrophy.

5. Read-through approach for stop mutations in Duchenne muscular dystrophy. An update.

Review 6. Clinical utility of serum biomarkers in Duchenne muscular dystrophy.

Review 7. Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy.

Review 8. Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy.