P.264SYROS study – long-term reduction in rate of respiratory function decline in patients with Duchenne muscular dystrophy treated with idebenone

2019 ◽  
Vol 29 ◽  
pp. S144
Author(s):  
L. Servais ◽  
O. Mayer ◽  
C. McDonald ◽  
C. Straathof ◽  
U. Schara ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Respiratory Function

scholarly journals Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy

2020 ◽  
Vol 30 (1) ◽  
pp. 5-16 ◽  
Author(s):  
Laurent Servais ◽  
Chiara S.M. Straathof ◽  
Ulrike Schara ◽  
Andrea Klein ◽  
Mika Leinonen ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Respiratory Function ◽  
Term Data

scholarly journals Idebenone as a Novel Therapeutic Approach for Duchenne Muscular Dystrophy

2015 ◽  
Vol 10 (2) ◽  
pp. 189 ◽  
Author(s):  
Gunnar M Buyse ◽  
Nuri Gueven ◽  
Craig M McDonald ◽  
◽  
◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Respiratory Function ◽  
Morbidity And Mortality ◽  
Phase Iii ◽  
Atp Production ◽  
Airway Clearance ◽  
Dystrophin Deficiency ◽  
Confirmatory Phase

Progressive loss of pulmonary function leads to early morbidity and mortality in Duchenne muscular dystrophy (DMD) due to both expiratory impairment with ineffective airway clearance, and inspiratory impairment leading to nocturnal and daytime hypoventilation and respiratory failure. Glucocorticoid steroids have become a mainstay of DMD therapy with well-documented efficacy on muscle strength and respiratory function. However, the side-effect profile restricts their long-term use, particularly in non-ambulant patients. Idebenone improves secondary mitochondrial dysfunction caused by dystrophin deficiency, intracellular calcium accumulation and increased reactive oxygen species (ROS). Idebenone-mediated improved bioenergetics leads to enhanced adenosine triphosphate (ATP) production and reduced ROS. Based on this rationale, idebenone has been investigated clinically for efficacy on reducing respiratory function decline in exploratory phase II (DELPHI) and confirmatory phase III (DELOS) trials. Idebenone significantly reduced the loss of respiratory function in 8–18-year-old DMD patients who were not using concomitant glucocorticoids. These results indicate that idebenone can modify the natural course of respiratory disease progression in DMD, which is relevant in clinical practice where loss of respiratory function continues to be a predominant cause of early morbidity and mortality in DMD.

scholarly journals Dental pulp stem cells can improve muscle dysfunction in animal models of Duchenne muscular dystrophy

2021 ◽  
Vol 12 (1) ◽  
Author(s):  
Yuko Nitahara-Kasahara ◽  
Mutsuki Kuraoka ◽  
Posadas Herrera Guillermo ◽  
Hiromi Hayashita-Kinoh ◽  
Yasunobu Maruoka ◽  
...  
Keyword(s):  
Stem Cells ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Dental Pulp ◽  
Systemic Treatment ◽  
Dental Pulp Stem Cells ◽  
Mri Findings ◽  
Systemic Injection ◽  
Therapeutic Benefits

Abstract Background Duchenne muscular dystrophy (DMD) is an inherited progressive disorder that causes skeletal and cardiac muscle deterioration with chronic inflammation. Dental pulp stem cells (DPSCs) are attractive candidates for cell-based strategies for DMD because of their immunosuppressive properties. Therefore, we hypothesized that systemic treatment with DPSCs might show therapeutic benefits as an anti-inflammatory therapy. Methods To investigate the potential benefits of DPSC transplantation for DMD, we examined disease progression in a DMD animal model, mdx mice, by comparing them with different systemic treatment conditions. The DPSC-treated model, a canine X-linked muscular dystrophy model in Japan (CXMDJ), which has a severe phenotype similar to that of DMD patients, also underwent comprehensive analysis, including histopathological findings, muscle function, and locomotor activity. Results We demonstrated a therapeutic strategy for long-term functional recovery in DMD using repeated DPSC administration. DPSC-treated mdx mice and CXMDJ showed no serious adverse events. MRI findings and muscle histology suggested that DPSC treatment downregulated severe inflammation in DMD muscles and demonstrated a milder phenotype after DPSC treatment. DPSC-treated models showed increased recovery in grip-hand strength and improved tetanic force and home cage activity. Interestingly, maintenance of long-term running capability and stabilized cardiac function was also observed in 1-year-old DPSC-treated CXMDJ. Conclusions We developed a novel strategy for the safe and effective transplantation of DPSCs for DMD recovery, which included repeated systemic injection to regulate inflammation at a young age. This is the first report on the efficacy of a systemic DPSC treatment, from which we can propose that DPSCs may play an important role in delaying the DMD disease phenotype.

Oral bisphosphonate treatment in patients with Duchenne muscular dystrophy on long term glucocorticoid therapy

2020 ◽  
Vol 30 (7) ◽  
pp. 599-610
Author(s):  
Cuixia Tian ◽  
Brenda L. Wong ◽  
Lindsey Hornung ◽  
Jane C. Khoury ◽  
Irina Rybalsky ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Glucocorticoid Therapy ◽  
Bisphosphonate Treatment ◽  
Oral Bisphosphonate

Long-term follow-up of dysphagia in adult patients with Duchenne muscular dystrophy

2018 ◽  
Vol 22 (5) ◽  
pp. 786-790 ◽  
Author(s):  
Yuka Yamada ◽  
Michiyuki Kawakami ◽  
Ayako Wada ◽  
Shogo Fukui ◽  
Koshiro Haruyama ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Adult Patients ◽  
Long Term Follow Up
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