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Public Health Genomics and Precision Health Knowledge Base (v9.0)
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Precision Health Database|Search|Public Health Genomics and Precision Health Knowledge Base (PHGKB)
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Last data update: Apr 25, 2024
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Duchenne Muscular Dystrophy[original query]>>Reviews/Commentaries[Product Type]
The Early Care (0-3 Years) In Duchenne Muscular Dystrophy Meeting Report.
Niki Armstrong et al. J Neuromuscul Dis 2024
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Detecting early signs in Duchenne muscular dystrophy: comprehensive review and diagnostic implications.
Eugenio Mercuri et al. Front Pediatr 2023 111276144
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Public Health Genetics, Gene Therapy, and Duchenne Muscular Dystrophy
CDC Webinar, December 18, 2023
For Duchenne moms who pushed for cures, new breakthrough therapy can’t rebuild what’s lost
J Mast, Stat News, June 30, 2023
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond.
Dongsheng Duan et al. Hum Gene Ther 2023 34(9-10) 345-349
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The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European Experience.
Annemieke Aartsma-Rus et al. Journal of neuromuscular diseases 2023
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Newborn Screening for the Diagnosis and Treatment of Duchenne Muscular Dystrophy.
Farrar Michelle A et al. Journal of neuromuscular diseases 2022
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Gene Therapy for Duchenne Muscular Dystrophy: Unlocking the Opportunities in Countries in the Middle East and Beyond.
Elbashir Haitham et al. Journal of neuromuscular diseases 2022
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CRISPR-Based Therapeutic Gene Editing for Duchenne Muscular Dystrophy: Advances, Challenges and Perspectives.
Chen Guofang et al. Cells 2022 11(19)
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Food and Drug Administration Lifts Clinical Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Linked to Patient Death.
Philippidis Alex et al. Human gene therapy 2022 33(11-12) 573-576
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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
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Genetic correction strategies for Duchenne Muscular Dystrophy and their impact on the heart.
Johnston Jamie R et al. Progress in pediatric cardiology 2021 63
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Genetic neuromuscular disorders: what is the best that we can do?
Laing Nigel G et al. Neuromuscular disorders : NMD 2021 31(10) 1081-1089
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Gene therapy for neuromuscular disorders: prospects and ethics.
Ryan Monique M et al. Archives of disease in childhood 2021
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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
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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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Overview of Gene Therapy in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy.
Abreu Nicolas J et al. Pediatric pulmonology 2020 Sep
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[Clinical Ethics in Hereditary Neuromuscular Diseases such as Duchenne Muscular Dystrophy].
Nakajima Takashi et al. Brain and nerve = Shinkei kenkyu no shinpo 2020 Jul 72(7) 753-766
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Duchenne Muscular Dystrophy- Where Genetic Testing is Inevitable and Vital!
Gupta Ambika et al. Indian journal of pediatrics 2020 May
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Is it the right time for an infant screening for Duchenne muscular dystrophy?
Vita Gian Luca et al. Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology 2020 Feb
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Clinical Development on the Frontier: Gene Therapy for Duchenne Muscular Dystrophy.
Asher Damon R et al. Expert opinion on biological therapy 2020 Feb
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Duchenne muscular dystrophy
H Fox et al, BMJ, January 2020
CRISPR enters its first human clinical trials- The gene editor targets cancer, blood disorders and blindness
T Hesman, Science News, August 14, 2019
This Drug Will Save Children's Lives. It Costs $2 Million.
NY Times editorial board, August 13, 2019
A mini-review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy.
Landfeldt Erik et al. Acta paediatrica (Oslo, Norway : 1992) 2019 Feb 108(2) 224-230
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Is Molecular Diagnosis Necessary for Children with Duchenne Muscular Dystrophy?
Puri Ratna Dua et al. Indian pediatrics 2019 Jul 56(7) 549-550
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Living with Muscular Dystrophy
CDC, 2019
New pharmacotherapies for genetic neuromuscular disorders: opportunities and challenges.
Ricci Federica et al. Expert review of clinical pharmacology 2019 Jun
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Maximizing the Benefit of Life-Saving Treatments for Pompe Disease, Spinal Muscular Atrophy, and Duchenne Muscular Dystrophy Through Newborn Screening: Essential Steps.
Baker Mei et al. JAMA neurology 2019 May
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[Application of next-generation sequencing in the molecular diagnosis of Duchenne muscular dystrophy].
Tian Pei-Chao et al. Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics 2019 Mar 21(3) 244-248
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Page last reviewed:
Feb 1, 2024
Page last updated:
Apr 25, 2024
Content source:
Public Health Genomics Branch in the Division of Blood Disorders and Public Health Genomics
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National Center on Birth Defects and Developmental Disabilities
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