Longitudinal data of serum creatine kinase levels and motor, pulmonary, and cardiac functions in 337 patients with Duchenne muscular dystrophy
- Published on 03/29/2024
- Reading time: 5 min.
Hiroyuki Awano MD, PhD 1, Yoshinori Nambu MD 2, Chieko Itoh BHS 3, Akihiro Kida 3, Tetsushi Yamamoto PhD 4, Tomoko Lee MD, PhD 5, Yasuhiro Takeshima MD, PhD 5, Kandai Nozu MD, PhD 2, Masafumi Matsuo MD, PhD 6
1
Research Initiative Center, Organization for Research Initiative and Promotion
Tottori University
Yonago
Japan
2
Department of Pediatrics
Kobe University Graduate School of Medicine
Kobe
Japan
3
Division of Rehabilitation Medicine
Kobe University Hospital
Kobe
Japan
4
Nagahama Institute of Bio‐Science and Technology
Nagahama
Japan
5
Department of Pediatrics
Hyogo Medical University
Nishinomiya
Japan
6
Faculty of Health Sciences
Kobe Tokiwa University
Kobe
Japan
Abstract
Introduction/Aims Duchenne muscular dystrophy (DMD) presents with skeletal muscle weakness, followed by cardiorespiratory involvement. The need for longitudinal data regarding DMD that could serve as a control for determining treatment efficacy in clinical trials has increased notably. The present study examined the longitudinal data of Japanese DMD patients collectively and assessed individual patients with pathogenic variants eligible for exon‐skipping therapy.
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