Expert council resolution on the use of pathogenetic therapy with Ataluren in patients with non-ambulatory Duchenne muscular dystrophy-Reference-Cited by-同舟云学术

Expert council resolution on the use of pathogenetic therapy with Ataluren in patients with non-ambulatory Duchenne muscular dystrophy

Published:2023-01-15 Issue:4 Volume:3 Page:148-151
ISSN:2712-794X
Container-title:L.O. Badalyan Neurological Journal
language:
Short-container-title:Nevrol. ž. im. L.O. Badalâna

Author:

Artemyeva Svetlana B.¹,Vlodavets Dmitry V.²,Guzeva Valentina I.³,Dzhaksybayeva Altynshash K.⁴,Zhauniaronak Irina V.⁵,Kuzenkova Ludmila M.⁶,Lepessova Marzhan M.⁷,Myrzalieva Bakhyt D.⁸,Pechatnikova Natalia L.⁹,Podkletnova Tatyana V.¹⁰,Suslov Vasily M.¹¹,Pelipenko Irina V.¹²,Bello Luca¹³,Butoianu Niculina¹⁴

Affiliation:

1. Pirogov Russian National Research University

2. Russian Children’s Neuromuscular Center; Research Clinical Institute of Pediatrics and Pediatric Surgery named after Academician Yu.E. Veltischev; Pirogov Russian National Research University

3. St. Petersburg State Pediatric Medical University of the Ministry of Health of Russia; Russian Society of Neurologists

4. Astana Medical University

5. Belarusian Medical Academy of Postgraduate Education

6. Centre for Children’s Psychoneurology; National Medical Research Centre for Children’s Health of the Ministry of Health of the Russian Federation; Clinical Institute of Children’s Health named after N.F. Filatov of the First Moscow State Medical University named after I.M. Sechenov of the Ministry of Health of Russia (Sechenov University)

7. Kazakh-Russian Medical University; Associations of Pediatric Neurologists of the Republic of Kazakhstan

8. Kazakh-Russian Medical University; Public Health Department

9. Orphan Center at the Morozov Clinical Hospital in Moscow

10. National Medical Research Centre for Children’s Health of the Ministry of Health of the Russian Federation

11. St. Petersburg State Pediatric Medical University

12. Regional Children’s Clinical Hospital of the Health Committee of the Kursk Region

13. University of Padova

14. Carol Davila University of Medicine and Pharmacy

Publisher

National Medical Research Center for Childrens Health

Subject

General Medicine

Reference11 articles.

1. Ebrahimi-Fakhari D., Dillmann U., Flotats-Bastardas M., Poryo M., Abdul-Khaliq H., Shamdeen M.G., et al. Off-label use of ataluren in four non-ambulatory patients with Duchenne muscular dystrophy: effects on cardiac and pulmonary function and muscle strength. Front. Pediatr. 2018; 6: 316. https://doi.org/10.3389/fped.2018.00316

2. McDonald C.M., Muntoni F., Penematsa V., Jiang J., Kristensen A., Bibbiani F., et al. Tulinius. Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients. J. Comp. Eff. Res. 2022; 11(3): 139–55. https://doi.org/10.2217/cer-2021-0196

3. Matthews E., Brassington R., Kuntzer T., Jichi F., Manzur A.Y. Corticosteroids for the treatment of Duchenne muscular dystrophy. Cochrane Database Syst. Rev. 2016; 2016(5): CD003725. https://doi.org/10.1002/14651858.cd003725.pub4

4. Lue Y.J., Lin R.F., Chen S.S., Lu Y.M. Measurement of the functional status of patients with different types of muscular dystrophy. Kaohsiung J. Med. Sci. 2009; 25(6): 325–33. https://doi.org/10.1016/s1607-551x(09)70523-6

5. Gandolla M., Antonietti A., Longatelli V., Biffi E., Diella E., Delle Fave M., et al. Test-retest reliability of the Performance of Upper Limb (PUL) module for muscular dystrophy patients. PLoS One. 2020; 15(9): e0239064. https://doi.org/10.1371/journal.pone.0239064