Cost of illness for neuromuscular diseases in the United States.
Larkindale J, Yang W, Hogan PF, et al. Cost of illness for neuromuscular diseases in the United States. Muscle &…
Health care utilization and costs for children and adults with duchenne muscular dystrophy.
Teoh LJ, Geelhoed EA, Bayley K, Leonard H, Laing NG. Health care utilization and costs for children and adults with…
The Direct Cost of Managing a Rare Disease: Assessing Medical and Pharmacy Costs Associated with Duchenne Muscular Dystrophy in the United States
Thayer S, Bell C, McDonald CM. The Direct Cost of Managing a Rare Disease: Assessing Medical and Pharmacy Costs Associated…
Social/economic costs and health-related quality of life in patients with Duchenne muscular dystrophy in Europe
Cavazza M, Kodra Y, Armeni P, et al. Social/economic costs and health-related quality of life in patients with Duchenne muscular…
An overview of Compassionate Use Programs in the European Union member states
Balasubramanian G, Morampudi S, Chhabra P, Gowda A, Zomorodi B. An overview of Compassionate Use Programs in the European Union…
The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review
Ryder S, Leadley RM, Armstrong N, et al. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence…
The New Paradigms in Clinical Research: From Early Access Programs to the Novel Therapeutic Approaches for Unmet Medical Needs
Scavone C, di Mauro G, Mascolo A, Berrino L, Rossi F, Capuano A. The New Paradigms in Clinical Research: From…
Patient views on research use of clinical data without consent: Legal, but also acceptable?
Richter G, Borzikowsky C, Lieb W, Schreiber S, Krawczak M, Buyx A. Patient views on research use of clinical data…
FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
Aartsma-Rus A, Krieg AM. FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga. Nucleic acid…
Extending Life in Duchenne Muscular Dystrophy: Implications for Appraisals of Cost-Effectiveness
Landfeldt E. Extending Life in Duchenne Muscular Dystrophy: Implications for Appraisals of Cost-Effectiveness. PharmacoEconomics - open 2019;3:279-280
