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ISHIGAKI KEIKO
Department School of Medicine(Tokyo Women's Medical University Hospital), School of Medicine Position Associate Professor |
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| Article types | Original article |
| Language | English |
| Peer review | Non peer reviewed |
| Title | Efficacy of steroid therapy for Fukuyama congenital muscular dystrophy. |
| Journal | Formal name：Scientific reports Abbreviation：Sci Rep ISSN code：20452322/20452322 |
| Domestic / Foregin | Foregin |
| Volume, Issue, Page | 11(1),pp.24229 |
| Author and coauthor | MURAKAMI Terumi, SATO takatoshi, ADACHI Michiru, ISHIGURO Kumiko, SHICHIJI Minobu, TACHIMORI Hisateru, NAGATA Satoru, ISHIGAKI KEIKO† |
| Authorship | Corresponding author |
| Publication date | 2021/12/20 |
| Summary | Although there is only symptomatic treatment for Fukuyama congenital muscular dystrophy (FCMD), several reports have suggested that steroid therapy could be effective for FCMD; however, no independent intervention studies have been conducted. This study aimed to evaluate the efficacy of steroid therapy for restoring motor functions in FCMD patients. This study involved 3-to-10-year-old FCMD patients who exhibited a decline in motor functions, requested steroid therapy. Patients with consent started oral administration of 0.5-mg/kg prednisolone every alternate day, which was increased to 1.0 mg/kg if the response was inadequate. We used the Gross Motor Function Measure (GMFM) to evaluate and compare the motor functions of all patients. Wilcoxon signed-rank test (significance level, P ≤ 0.05) was used for statistical analysis. At the onset of steroid therapy, 8.10 years (SD, 2.14 years) was the mean age of FCMD patients. The mean GMFM difference between before and after the steroid therapy was + 1.23 (SD, 1.10), and a P value of 0.015 represented significant improvement in GMFM. Our results indicate that steroid therapy may contribute to the maintenance and improvement of the motor functions of advanced-stage FCMD patients.Clinical Trial Registration Registration Number: UMIN000020715, Registration Date: Feb 1st, 2016 (01/02/2016). |
| DOI | 10.1038/s41598-021-03781-z |
| PMID | 34930981 |