Latest Advances

  • Condition: Adolescent Boys with Duchenne Muscular Dystrophy (DMD)
  • Journal: European journal of endocrinology
  • Treatment Used: Pubertal Induction
  • Number of Patients: 0
  • Published —
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 —
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 —
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 —
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 —
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 —
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 —
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 —
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 —
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 —
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 —
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 —
In this study, researchers evaluated the effects of scoliosis surgery on the lung function of patients with muscular dystrophies or spinal muscular atrophy.
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