Warning: Undefined array key "mm" in /www/wwwroot/www.ai-bt.com/si.php on line 10 Deprecated: trim(): Passing null to parameter #1 ($string) of type string is deprecated in /www/wwwroot/www.ai-bt.com/si.php on line 10 European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene.

Literature DB >> 25497400

European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene.

Manuel Haas¹, Viktor Vlcek², Pavel Balabanov², Tomas Salmonson³, Serge Bakchine⁴, Greg Markey⁵, Martina Weise⁶, Gabriele Schlosser-Weber⁶, Henning Brohmann⁶, Concepcion Prieto Yerro⁷, Macarena Rodriguez Mendizabal⁷, Violeta Stoyanova-Beninska⁸, Hans L Hillege⁸.

Abstract

Entities: Disease Gene Species

Mesh：

Substances：

Year: 2014 PMID： 25497400 DOI： 10.1016/j.nmd.2014.11.011

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

Keyword Cloud
Cited

× No keyword cloud information.

55 in total

Review 1. Gene-targeting pharmaceuticals for single-gene disorders.

Authors: Arthur L Beaudet; Linyan Meng
Journal: Hum Mol Genet Date: 2015-11-30 Impact factor: 6.150

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

Authors: Laure Bidou; Olivier Bugaud; Valery Belakhov; Timor Baasov; Olivier Namy
Journal: RNA Biol Date: 2017-02-01 Impact factor: 4.652

3. Premature termination codons in the DMD gene cause reduced local mRNA synthesis.

Authors: Raquel García-Rodríguez; Monika Hiller; Laura Jiménez-Gracia; Zarah van der Pal; Judit Balog; Kevin Adamzek; Annemieke Aartsma-Rus; Pietro Spitali
Journal: Proc Natl Acad Sci U S A Date: 2020-07-02 Impact factor: 11.205

4. Oligonucleotide therapies for disorders of the nervous system.

Authors: Olga Khorkova; Claes Wahlestedt
Journal: Nat Biotechnol Date: 2017-02-27 Impact factor: 54.908

5. A New Kid on the Playground of CRISPR DMD Therapy.

Authors: Dongsheng Duan
Journal: Hum Gene Ther Clin Dev Date: 2017-06 Impact factor: 5.032

6. Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression.

Authors: Bijoyita Roy; Westley J Friesen; Yuki Tomizawa; John D Leszyk; Jin Zhuo; Briana Johnson; Jumana Dakka; Christopher R Trotta; Xiaojiao Xue; Venkateshwar Mutyam; Kim M Keeling; James A Mobley; Steven M Rowe; David M Bedwell; Ellen M Welch; Allan Jacobson
Journal: Proc Natl Acad Sci U S A Date: 2016-10-04 Impact factor: 11.205

7. Longitudinal metabolomic analysis of plasma enables modeling disease progression in Duchenne muscular dystrophy mouse models.

Authors: Roula Tsonaka; Mirko Signorelli; Ekrem Sabir; Alexandre Seyer; Kristina Hettne; Annemieke Aartsma-Rus; Pietro Spitali
Journal: Hum Mol Genet Date: 2020-03-27 Impact factor: 6.150