Duchenne Muscular Dystrophy
Last Posted: Apr 01, 2021
- Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy.
Peay Holly L et al. Molecular genetics & genomic medicine 2021 e1664
- Consensus Statement on the Management of Duchenne Muscular Dystrophy in Saudi Arabia During the Coronavirus Disease 2019 Pandemic.
Bamaga Ahmed K et al. Frontiers in pediatrics 2021 9629549
- Duchenne muscular dystrophy.
Duan Dongsheng et al. Nature reviews. Disease primers 2021 Feb 7(1) 13
- Newborn screening of duchenne muscular dystrophy specifically targeting deletions amenable to exon-skipping therapy.
Beckers Pablo et al. Scientific reports 2021 Feb 11(1) 3011
- Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy
V Himic et al, EJHG, February 9, 2021
- Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy
RJ Wilcocks et al, JAMA Network Open, January 4, 2021
- Current Clinical Applications of in vivo Gene Therapy with AAVs.
Mendell Jerry R et al. Molecular therapy : the journal of the American Society of Gene Therapy 2020 Dec
- Genotype-Phenotype Correlations in Duchenne and Becker Muscular Dystrophy Patients from the Canadian Neuromuscular Disease Registry.
Lim Kenji Rowel Q et al. Journal of personalized medicine 2020 Nov 10(4)
- Duchenne Muscular Dystrophy Newborn Screening, a Case Study for Examining Ethical and Legal Issues for Pilots for Emerging Disorders: Considerations and Recommendations.
Lloyd-Puryear Michele A et al. International journal of neonatal screening 2018 Mar 4(1) 6
- Overview of Gene Therapy in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy.
Abreu Nicolas J et al. Pediatric pulmonology 2020 Sep
Rare Disease PHGKB is an online, continuously updated, searchable database of published scientific literature, CDC and NIH resources, and other information that address the public health impact and translation of genomic and other precision health discoveries into improved health outcomes related to rare diseases...more
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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
- Graves Disease
- Huntington Disease
- Myasthenia Gravis
- Retinitis Pigmentosa
- Severe Combined Immunodeficiency
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