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
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