Low serum cholic acid concentration of Duchenne muscular dystrophy

Tanaka, Kaoru; Takeshita, Kenzo; Suganuma, Ikuo; Kasagi, Shigeto

doi:10.1016/s0387-7604(83)80085-0

Cited by 2 publications

(2 citation statements)

References 8 publications

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“…There were no apparent consistent changes in serum lipid and cholate biomarkers from Day 1 predose to Day 11 predose. Serum concentrations observed for deoxycholic acid, cholic acid and chenodeoxycholic acid were similar to the low levels reported by Tanaka et al [ 12 ] for DMD patients compared with controls; although, the ages of the DMD patients who participated in this study were generally lower. Interpretation of the data was difficult with only one timepoint postdose and no placebo-control group.…”

Section: Discussionsupporting

confidence: 89%

Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

Ricotti¹,

Spinty

Roper

et al. 2016

PLoS ONE

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PurposeSMT C1100 is a utrophin modulator being evaluated as a treatment for Duchenne muscular dystrophy (DMD). This study, the first in pediatric DMD patients, reports the safety, tolerability and PK parameters of single and multiple doses of SMT C1100, as well as analyze potential biomarkers of muscle damage.MethodsThis multicenter, Phase 1 study enrolled 12 patients, divided equally into three groups (A–C). Group A were given 50 mg/kg on Days 1 and 11, and 50 mg/kg bid on Days 2 to 10. Group B and C received 100 mg/kg on Days 1 and 11; Group B and Group C were given 100 mg/kg bid and 100 mg/kg tid, respectively, on Days 2 to 10. A safety review was performed on all patients following the single dose and there was at least 2 weeks between each dose escalation, for safety and PK review. Adverse events (AEs) were monitored throughout the study.ResultsMost patients experienced mild AEs and there were no serious AEs. Two patients required analgesia for pain (headache, ear pain and toothache). One patient experienced moderate psychiatric AEs (abnormal behaviour and mood swings). Plasma concentrations of SMT C1100 at Days 1 and 11 indicated a high degree of patient variability regardless of dose. Unexpectedly the SMT C1100 levels were significantly lower than similar doses administered to healthy volunteers in an earlier clinical study. In general, individual baseline changes of creatine phosphokinase, alanine aminotransferase, aspartate aminotransferase levels fell with SMT C1100 dosing.ConclusionsSMT C1100 was well tolerated in pediatric DMD patients.Trial RegistrationClinicalTrials.gov NCT02383511

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Section: Discussionsupporting

confidence: 89%

Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

Ricotti¹,

Spinty

Roper

et al. 2016

PLoS ONE

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“…Study characteristics are described in Table 1. A total of 1,865 individuals afflicted by different types of MDs were included, and sample size ranged from 15 to 829 individuals [32][33][34][35][36][37][38][39][40][41][42][43][44][45][46][47][48]. All included studies were cross-sectional.…”

Section: Study Characteristicsmentioning

confidence: 99%

Dyslipidemia in Muscular Dystrophy: A Systematic Review and Meta-Analysis

Sun

Wang

White

et al. 2023

Journal of Neuromuscular Diseases

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Background: Muscular dystrophies (MDs) are characterized by chronic muscle wasting but also poorly understood metabolic co-morbidities. We have recently shown that Duchenne MD (DMD) patients, dogs and asymptomatic carriers are affected by a new form of dyslipidemia that may exacerbate muscle damage. Objective: We aimed to perform a systematic review and meta-analysis for evidence that other types of MDs are associated with dyslipidemia compared to healthy controls. Methods: Search was conducted using MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials for reports that compare plasma/serum lipids from MD patients and controls, and meta-analysis of cross-sectional studies quantifying total cholesterol, high-density lipoprotein, low density lipoprotein and triglycerides was performed. Results: Out of 749 studies, 17 met our inclusion criteria for meta-analysis. 14 of the 17 studies (82% ) included investigated myotonic dystrophy (DM); other studies were on pseudohypertrophic MD (PMD) or DMD. As a whole, MD individuals had significantly higher levels of circulating total cholesterol (Hedges’ g with 95% confidence interval [CI], 0.80 [0.03 – 1.56]; p = 0.04) and triglycerides (Hedges’ g with 95% confidence interval [CI], 2.28[0.63 – 3.92]; p = 0.01) compared to controls. Meta-regression analysis showed the percentage of male gender was significantly associated with the difference in total cholesterol (beta = 0.05; 95% CI, – 0.02 to 0.11; p = 0.043) and high-density lipoprotein (beta = – 9.38; 95% CI, – 16.26 to – 2.50; p = 0.028). Conclusions: MD is associated with significantly higher circulating levels of total cholesterol and triglycerides. However, caution on the interpretation of these findings is warranted and future longitudinal research is required to better understand this relationship.

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Low serum cholic acid concentration of Duchenne muscular dystrophy

Cited by 2 publications

References 8 publications

Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

Dyslipidemia in Muscular Dystrophy: A Systematic Review and Meta-Analysis

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