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Latest Advances in
Becker Muscular Dystrophy
Overview
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Clinical Trials
Latest Advances
Condition:
Adolescent Boys with Duchenne Muscular Dystrophy (DMD)
Journal:
European journal of endocrinology
Treatment Used:
Pubertal Induction
Number of Patients:
0
Published —
October 28, 2020
This study determined the effects of pubertal induction in adolescent boys with Duchenne muscular dystrophy (DMD).
Condition:
Duchenne Muscular Dystrophy Patients with Left Ventricular Dysfunction
Journal:
European review for medical and pharmacological sciences
Treatment Used:
Sacubitril-Valsartan
Number of Patients:
3
Published —
September 23, 2020
The purpose of the study was to assess whether treatment with sacubitril/valsartan is well tolerated and may have beneficial effects in Duchenne Muscular Dystrophy patients with left ventricle dysfunction.
Condition:
Duchenne Muscular Dystrophy
Journal:
PLoS medicine
Treatment Used:
Vamorolone
Number of Patients:
23
Published —
September 21, 2020
This study tested the safety and efficacy of using vamorolone to treat patients with Duchenne muscular dystrophy.
Condition:
Chronic Granulomatous Disease (CGD) with the McLeod Phenotype in France
Journal:
Journal of clinical immunology
Treatment Used:
Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Number of Patients:
8
Published —
June 21, 2020
This study described French patients with chronic granulomatous disease (CGD) and the McLeod phenotype.
Condition:
Duchenne Muscular Dystrophy (DMD)
Journal:
Pharmacological research
Treatment Used:
Nutritional Supplements
Number of Patients:
0
Published —
June 03, 2020
This article discusses safety issues and harmful pharmacological interactions of nutritional supplements in Duchenne muscular dystrophy.
Condition:
Inherited Neuromuscular Diseases of Childhood
Journal:
Seminars in neurology
Treatment Used:
Targeted Treatments
Number of Patients:
0
Published —
April 16, 2020
This review highlights advances and emerging targeted therapies for Duchenne muscular dystrophy and centronuclear myopathy, while also covering enzyme replacement therapy and small molecule-based targeted therapies for conditions such as Pompe's disease and congenital myasthenic syndromes.
Condition:
Muscular Dystrophy (MD)
Journal:
Circulation. Heart failure
Treatment Used:
Heart Transplantation (HTx)
Number of Patients:
122
Published —
March 29, 2020
This study described heart transplantation (HTx) outcomes for patients with muscular dystrophy (MD).
Condition:
Duchenne Muscular Dystrophy and Spinal Muscular Atrophy
Journal:
The American journal of occupational therapy : official publication of the American Occupational Therapy Association
Treatment Used:
TheraBite® Device
Number of Patients:
3
Published —
March 25, 2020
In this study, researchers evaluated the effectiveness of the new TheraBite® device on the oral range of motion of people with Duchenne muscular dystrophy and spinal muscular atrophy
Condition:
Duchenne muscular dystrophy
Journal:
Neurology
Treatment Used:
Golodirsen
Number of Patients:
12
Published —
March 07, 2020
The study researched the outcomes of Golodirsen in patients with Duchenne muscular dystrophy.
Condition:
Duchenne Muscular Dystrophy
Journal:
Muscle & nerve
Treatment Used:
Recombinant Human Insulin-Like Growth Factor-1 (rhIGF) Therapy
Number of Patients:
38
Published —
February 29, 2020
This study tested the safety and efficacy of using rhIGF therapy to treat children with Duchenne muscular dystrophy.
Condition:
Duchenne Muscular Dystrophy
Journal:
The Annals of pharmacotherapy
Treatment Used:
Deflazacort
Number of Patients:
0
Published —
February 06, 2020
In this study, researchers evaluated the effectiveness of deflazacort for the treatment of Duchenne muscular dystrophy.
Condition:
Muscular Dystrophies and Spinal Muscular Atrophy
Journal:
Pediatric pulmonology
Treatment Used:
Scoliosis Surgery
Number of Patients:
20
Published —
February 04, 2020
In this study, researchers evaluated the effects of scoliosis surgery on the lung function of patients with muscular dystrophies or spinal muscular atrophy.
Showing 1-12 of 42
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