Universität zu Köln

Krosschell, Kristin J., Kissel, John T., Townsend, Elise L., Simeone, Sarah D., Zhang, Ren Zhe, Reyna, Sandra P., Crawford, Thomas O., Schroth, Mary K., Acsadi, Gyula, Kishnani, Priya S., Von Kleist-Retzow, Juergen-Christoph, Hero, Barbara, D'Anjou, Guy, Smith, Edward C., Elsheikh, Bakri, Simard, Louise R., Prior, Thomas W., Scott, Charles B., Lasalle, Bernard, Sakonju, Ai, Wirth, Brunhilde ORCID: 0000-0003-4051-5191 and Swoboda, Kathryn J. (2018). CLINICAL TRIAL OF L-CARNITINE AND VALPROIC ACID IN SPINAL MUSCULAR ATROPHY TYPE I. Muscle Nerve, 57 (2). S. 193 - 200. HOBOKEN: WILEY. ISSN 1097-4598

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Abstract

Introduction: The aim of this study was to determine the safety and therapeutic potential of L-carnitine and valproic acid (VPA) in infants with spinal muscular atrophy (SMA). Methods: Our investigation was an open-label phase 2 multicenter trial of L-carnitine and VPA in infants with SMA type I with retrospective comparison to an untreated, matched cohort. Primary outcomes were: safety and adverse events; secondary outcomes were survival, time to death/> 16 hours/day of ventilator support; motor outcomes; and maximum ulnar compound motor action potential amplitude. Results: A total of 245 AEs were observed in 35 of the 37 treated subjects (95%). Respiratory events accounted for 49% of all adverse events, resulting in 14 deaths. Survival was not significantly different between treated and untreated cohorts. Discussion: This trial provides evidence that, in infants with SMA type I, L-carnitine/VPA is ineffective at altering survival. The substantial proportion of infants reaching end-points within 6 months of enrollment underscores the urgent need for pre-symptomatic treatment in SMA type I.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Krosschell, Kristin J. UNSPECIFIED UNSPECIFIED UNSPECIFIED Kissel, John T. UNSPECIFIED UNSPECIFIED UNSPECIFIED Townsend, Elise L. UNSPECIFIED UNSPECIFIED UNSPECIFIED Simeone, Sarah D. UNSPECIFIED UNSPECIFIED UNSPECIFIED Zhang, Ren Zhe UNSPECIFIED UNSPECIFIED UNSPECIFIED Reyna, Sandra P. UNSPECIFIED UNSPECIFIED UNSPECIFIED Crawford, Thomas O. UNSPECIFIED UNSPECIFIED UNSPECIFIED Schroth, Mary K. UNSPECIFIED UNSPECIFIED UNSPECIFIED Acsadi, Gyula UNSPECIFIED UNSPECIFIED UNSPECIFIED Kishnani, Priya S. UNSPECIFIED UNSPECIFIED UNSPECIFIED Von Kleist-Retzow, Juergen-Christoph UNSPECIFIED UNSPECIFIED UNSPECIFIED Hero, Barbara UNSPECIFIED UNSPECIFIED UNSPECIFIED D'Anjou, Guy UNSPECIFIED UNSPECIFIED UNSPECIFIED Smith, Edward C. UNSPECIFIED UNSPECIFIED UNSPECIFIED Elsheikh, Bakri UNSPECIFIED UNSPECIFIED UNSPECIFIED Simard, Louise R. UNSPECIFIED UNSPECIFIED UNSPECIFIED Prior, Thomas W. UNSPECIFIED UNSPECIFIED UNSPECIFIED Scott, Charles B. UNSPECIFIED UNSPECIFIED UNSPECIFIED Lasalle, Bernard UNSPECIFIED UNSPECIFIED UNSPECIFIED Sakonju, Ai UNSPECIFIED UNSPECIFIED UNSPECIFIED Wirth, Brunhilde UNSPECIFIED orcid.org/0000-0003-4051-5191 UNSPECIFIED Swoboda, Kathryn J. UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-198243
DOI:	10.1002/mus.25776
Journal or Publication Title:	Muscle Nerve
Volume:	57
Number:	2
Page Range:	S. 193 - 200
Date:	2018
Publisher:	WILEY
Place of Publication:	HOBOKEN
ISSN:	1097-4598
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language PERIPHERAL-NERVES; REFERENCE VALUES; UPPER EXTREMITY; RELIABILITY; ULTRASOUND; ULTRASONOGRAPHY; NEUROPATHIES Multiple languages Clinical Neurology; Neurosciences Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/19824