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

T.P.51 Metformin reduces weight and BMI in Duchenne muscular dystrophy patients on long term glucocorticoid therapy

2012 ◽  
Vol 22 (9-10) ◽  
pp. 866 ◽  
Author(s):  
S.E. Weatherspoon ◽  
J. Collins ◽  
H. Sucharew ◽  
B.L. Wong ◽  
I. Rybalsky ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Glucocorticoid Therapy

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.

P4.1 Puberty is delayed or absent in Duchenne Muscular Dystrophy boys on chronic glucocorticoid therapy

2011 ◽  
Vol 21 (9-10) ◽  
pp. 704
Author(s):  
M.M. Rutter ◽  
S.R. Rose ◽  
J. Collins ◽  
H. Sucherew ◽  
B.L. Wong ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Glucocorticoid Therapy

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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