Last Posted: Sep 15, 2021
- Muscular Dystrophy
- Gene therapy for neuromuscular disorders: prospects and ethics.
Ryan Monique M et al. Archives of disease in childhood 2021
- Genetic identification of pathogenic variations of the DMD gene: a retrospective study from 10,481 neonatal patients based on next-generation sequencing data.
Xiao Tiantian et al. Annals of translational medicine 2021 9(9) 766
- The socioeconomic burden of facioscapulohumeral muscular dystrophy.
Blokhuis Anna M et al. Journal of neurology 2021
- Current Status of Gene Therapy Research in Polyglutamine Spinocerebellar Ataxias.
Afonso-Reis Ricardo et al. International journal of molecular sciences 2021 22(8)
- Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders.
Lochmüller Hanns et al. Orphanet journal of rare diseases 2021 16(1) 141
- 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
- Duchenne and Becker Muscular Dystrophies' Prevalence in MD STARnet Surveillance Sites: An Examination of Racial and Ethnic Differences.
Zhang Yanan et al. Neuroepidemiology 2021 Jan 1-9
- 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
- Elucidation of the Genetic Cause in Dutch Limb Girdle Muscular Dystrophy Families: A 27-Year's Journey.
Ten Dam L et al. Journal of neuromuscular diseases 2020 Dec
- Mutational Spectrum of CAPN3 with Genotype-Phenotype Correlations in Limb Girdle Muscular Dystrophy Type 2A/R1 (LGMD2A/LGMDR1) patients in India.
Pathak Pankaj et al. Journal of neuromuscular diseases 2020 Dec
- 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
- Clinical and Genomic Evaluation of 207 Genetic Myopathies in the Indian Subcontinent.
Chakravorty Samya et al. Frontiers in neurology 2020 11559327
- 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)
- Association of genetic mutations and loss of ambulation in childhood-onset dystrophinopathy.
Haber Gregory et al. Muscle & nerve 2020 Nov
- 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
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