Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I

Author:

Krosschell Kristin J.1,Kissel John T.2,Townsend Elise L.3,Simeone Sarah D.4,Zhang Ren Zhe4,Reyna Sandra P.5,Crawford Thomas O.6,Schroth Mary K.7,Acsadi Gyula8,Kishnani Priya S.9,Von Kleist-Retzow Jürgen-Christoph10,Hero Barbara10,D'Anjou Guy11,Smith Edward C.9,Elsheikh Bakri2,Simard Louise R.12,Prior Thomas W.13,Scott Charles B.14,Lasalle Bernard15,Sakonju Ai16,Wirth Brunhilde1718,Swoboda Kathryn J.4ORCID,

Affiliation:

1. Department of Physical Therapy and Human Movement Sciences, Feinberg School of Medicine; Northwestern University; Chicago Illinois USA

2. Department of Neurology; The Ohio State University Wexner Medical Center; Columbus Ohio USA

3. Department of Physical Therapy; Institute of Health Professions, Massachusetts General Hospital; Boston Massachusetts USA

4. Department of Neurology; Center for Genomic Medicine, Massachusetts General Hospital; 185 Cambridge Street, Simches 5-240, Boston Massachusetts 02114 USA

5. Department of Neurology; University of Utah; Salt Lake City Utah USA

6. Department of Neurology; Johns Hopkins University School of Medicine; Baltimore Maryland USA

7. Department of Pediatrics, School of Medicine and Public Health; University of Wisconsin; Madison Wisconsin USA

8. Connecticut Children's Medical Center; Hartford Connecticut USA

9. Department of Pediatrics; Duke University; Durham North Carolina USA

10. Department of Pediatrics; Hospital of the University of Cologne; Cologne Germany

11. Department of Pediatrics; Saint-Justine Hospital; Montreal Quebec Canada

12. Biochemistry and Medical Genetics; University of Manitoba; Winnepeg Manitoba Canada

13. Department of Molecular Pathology; The Ohio State University; Columbus Ohio USA

14. CBS Squared, Inc; Fort Washington Pennsylvania USA

15. Department of Bioinformatics; University of Utah; Salt Lake City Utah USA

16. Department of Neurology; State University of New York; Syracuse New York

17. Institute of Human Genetics, Center for Molecular Medicine Cologne, Center for Rare Diseases Cologne

18. Institute of Genetics; University of Cologne; Cologne Germany

Funder

Cure SMA

Bild “Ein Herz für Kinder”

Eunice Kennedy Shriver National Institutes of Health and Human Development

Publisher

Wiley

Subject

Physiology (medical),Cellular and Molecular Neuroscience,Neurology (clinical),Physiology

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