Duchenne Muscular Dystrophy Formulary Central

Clinical Impact of Disease Progression

An evidence-based event model for Duchenne muscular dystrophy in the United States

Characterizing the natural history of Duchenne muscular dystrophy in the United States in real-world commercial and medicaid data

The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review

The impact of genotype on outcomes in individuals with Duchenne muscular dystrophy: a systematic review

Economic Impact of Disease Progression

Disease progression stages and burden in patients with Duchenne muscular dystrophy using administrative claims supplemented by electronic medical records

Health-related quality of life and economic burden of Duchenne muscular dystrophy (DMD): a systematic literature review

Patient Quality of Life (QoL)

Factors associated with the health-related quality of life among people with Duchenne muscular dystrophy: a study using the Health Utilities Index (HUI)

The impact of key health state transitions on health-related quality of life in Duchenne muscular dystrophy: a qualitative study

Patient/Caregiver QoL

Characterizing the impact on work productivity in patients with DMD and caregivers: an economic analysis

Qualitative study to understand the Duchenne muscular dystrophy (DMD) experience from the caregiver/patient perspective

Societal Perspectives on QoL

QALYs and ambulatory status: societal preferences for healthcare decision making

Societal perspectives on disease and treatment attributes characterizing rare diseases: a qualitative study from the United States

Disease attributes most important from a societal perspective: a case study involving Duchenne muscular dystrophy

Societal perspectives on the importance of disease and treatment attributes: a qualitative study from the United States

Outcomes Assessments

The feasibility of using US claims data to assess outcomes in Duchenne muscular dystrophy

Value and Cost Assessments

Assessing the impact of single or short-term administration on a therapy's cost-effectiveness: a hypothetical disease-agnostic model

Commercial health plans use of patient subgroup restrictions: an analysis of orphan and US Food and Drug Administration-expedited programs

Determining the “severity premium” for Duchenne muscular dystrophy: a societal consideration for cost-effectiveness analysis

Manuscripts Permissions