Last Posted: Jun 13, 2024
- Subspecialty Health Care Utilization in Pediatric Patients With Muscular Dystrophy in the United States.
Susan E Matesanz et al. Neurol Clin Pract 2024 14(4) e200312 - Medicaid spending and utilization of gene and RNA therapies for rare inherited conditions.
Ilina C Odouard et al. Health Aff Sch 2024 2(5) qxae051 - Short developmental milestone risk assessment tool to identify Duchenne muscular dystrophy in primary care.
Paula van Dommelen et al. Orphanet J Rare Dis 2024 19(1) 192 - Assessing the Benefits and Harms Associated with Early Diagnosis from the Perspective of Parents with Multiple Children Diagnosed with Duchenne Muscular Dystrophy.
Oindrila Bhattacharyya et al. Int J Neonatal Screen 2024 10(2) - The FDA and Gene Therapy for Duchenne Muscular Dystrophy
- Newborn screening for Duchenne muscular dystrophy: the perspectives of stakeholders.
Charli Ji et al. Lancet Reg Health West Pac 2024 3 101049 - Diagnosis and Management of Myotonic Dystrophy Type 1
J Hartman et al, JAMA March 11, 2024 - Spending on Targeted Therapies for Duchenne Muscular Dystrophy
L Bendicksen et al, JAMA, March 11, 2024 - Detecting missed diagnoses of spinal muscular atrophy in genome, exome, and panel sequencing datasets.
Ben Weisburd et al. medRxiv 2024 - Diagnostic Accuracy of Creatine Kinase Isoenzyme-MM Test in Newborn Screening for Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis.
Liang Tang et al. Pediatr Neurol 2024 15384-91
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