Duchenne Muscular Dystrophy
Last Posted: Jun 27, 2022
- Incidence of Duchenne muscular dystrophy in the modern era; an Australian study
D Kariyawasam et al, EJHG, June 27, 2022
- Selected clinical and demographic factors and all-cause mortality among individuals with Duchenne muscular dystrophy in the Muscular Dystrophy Surveillance, Tracking, and Research Network.
Paramsothy Pangaja et al. Neuromuscular disorders : NMD 2022 5
- Disease Progression Stages and Burden in Patients with Duchenne Muscular Dystrophy Using Administrative Claims Supplemented by Electronic Medical Records.
Iff Joel et al. Advances in therapy 2022
- A Decade of Progress in Gene Targeted Therapeutic Strategies in Duchenne Muscular Dystrophy: A Systematic Review.
Chung Liang Lam et al. Frontiers in bioengineering and biotechnology 2022 10833833
- Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Guglieri Michela et al. JAMA 2022 4
- Time to Diagnosis of Duchenne Muscular Dystrophy Remains Unchanged: Findings from the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet), 2000-2015.
Thomas Shiny et al. Muscle & nerve 2022
- The case for screening in early life for 'non-treatable' disorders: ethics, evidence and proportionality. A report from the Health Council of the Netherlands.
Kalkman Shona et al. European journal of human genetics : EJHG 2022
- Genetic correction strategies for Duchenne Muscular Dystrophy and their impact on the heart.
Johnston Jamie R et al. Progress in pediatric cardiology 2021 63
- Use of a book to disclose a diagnosis of Duchenne muscular dystrophy to a young child: A pilot study.
Audic F et al. Archives de pediatrie : organe officiel de la Societe francaise de pediatrie 2021
- Clinical and Genetic Characteristics in Young, Glucocorticoid-Naive Boys With Duchenne Muscular Dystrophy.
Schiava Marianela et al. Neurology 2021
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Selected Rare Diseases
- Alpha-1 Antitrypsin Deficiency
- Amyotrophic Lateral Sclerosis
- Brugada Syndrome
- Cerebral Palsy
- Cystic Fibrosis
- Duchenne Muscular Dystrophy
- Erythema Multiforme
- Familial Mediterranean Fever
- Fragile X Syndrome
- Gaucher Disease
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- Huntington Disease
- Myasthenia Gravis
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