Decline in Respiratory Function and Experience with Long-Term Assisted Ventilation in Advanced Duchenne's Muscular Dystrophy

CHEST Journal ◽  
1990 ◽  
Vol 97 (4) ◽  
pp. 884-889 ◽  
Author(s):  
Ahmet Baydun ◽  
Irene Gilgoff ◽  
William Prentice ◽  
Michael Carlson ◽  
D. Armin Fischer
Keyword(s):  
Muscular Dystrophy ◽  
Respiratory Function ◽  
Assisted Ventilation ◽  
Duchenne's Muscular Dystrophy

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

Long-term electrocardiographic studies of patients with Duchenne's muscular dystrophy.

1988 ◽  
Vol 29 (3) ◽  
pp. 295-300 ◽  
Author(s):  
Suguru MATSUOKA ◽  
Yuji AKITA ◽  
Satomi KAWAHITO ◽  
Atsuko NISHIOKA ◽  
Yoko MIKI ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Duchenne's Muscular Dystrophy

scholarly journals Long-term observation of cardiac function in Duchenne's muscular dystrophy. Evaluation using systolic time intervals and echocardiography.

1990 ◽  
Vol 31 (5) ◽  
pp. 585-597 ◽  
Author(s):  
Toshinori UTSUNOMIYA ◽  
Hideki MORI ◽  
Noritoshi SHIBUYA ◽  
Yasuhiko OKU ◽  
Shuzo MATSUO ◽  
...  
Keyword(s):  
Muscular Dystrophy ◽  
Cardiac Function ◽  
Systolic Time Intervals ◽  
Time Intervals ◽  
Systolic Time ◽  
Long Term Observation ◽  
Duchenne's Muscular Dystrophy

Long-term Nasal Intermittent Positive Pressure Ventilation in Advanced Duchenne's Muscular Dystrophy

CHEST Journal ◽  
1994 ◽  
Vol 105 (2) ◽  
pp. 445-448 ◽  
Author(s):  
Andrea Vianello ◽  
Matteo Bevilacqua ◽  
Vittorino Salvador ◽  
Claudio Cardaioli ◽  
Ezio Vincenti
Keyword(s):  
Muscular Dystrophy ◽  
Positive Pressure Ventilation ◽  
Intermittent Positive Pressure Ventilation ◽  
Positive Pressure ◽  
Duchenne's Muscular Dystrophy

scholarly journals A CASE REPORT ON THE MANAGEMENT OF DUCHENNE’S MUSCULAR DYSTROPHY (DMD) WITH MATRA BASTI

2021 ◽  
Vol p5 (02) ◽  
pp. 2750-2755
Author(s):  
Sharayu Kachole ◽  
Niranjan Rao ◽  
Padmakiran C
Keyword(s):  
Muscular Dystrophy ◽  
Treatment Protocol ◽  
Dystrophin Gene ◽  
Shoulder Girdle ◽  
Recessive Mutation ◽  
Multiple Systems ◽  
Before And After ◽  
Dual Action ◽  
Duchenne's Muscular Dystrophy

Duchenne’s Muscular Dystrophy (DMD) is the most common x-linked disorder in children presenting in early childhood due to recessive mutation of the dystrophin gene. It is characterized by progressive weak-ness in hip and shoulder girdle muscles beginning by age 5. Although there is no absolute cure for DMD, therapies can delay the onset or slow down the progression of disease. Survival beyond the age of 30 years is rare due to the severe chronic complication involving multiple systems. Serum Creatine Phosphokinase (CPK) level determination is the most specific objective parameter for the diagnosis and assessment of se-verity of Muscular Dystrophy (MD). Based on the presentation of DMD, it can be diagnosed as Mamsavru-ta Vatal leading to Mamsa Dhatu Upashoshana which is the consequence of Beejabhagavikruti. There will be continuous destruction of Dhatu leading to Vataprakopa again. Hence, the line of management is con-trolling the Vata and preventing the destruction. The best treatment for it which has this dual action is Mat-ra Basti. With this basic concept, a male child of 10 years diagnosed as Mamsavruta Vata was treated with Matra Basti using Dhanwantara Taila 10 ml/day for about 9 months. As there was associated Sama Do-shavastha, Agnichikitsa Lepa was advised for 7 days initially. The treatment showed significant reduction in the level of CPK tested before and after the treatment. Hence, the treatment protocol followed can be taken up for further research and can prove with evidence that, it can be effectively practiced in the man-agement of DMD. The long-term benefit, improvement in disease, quality of life can be accessed through the research with long term follow-ups.

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.

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