B. Maru

The clinical, economic, and humanistic burden of Dravet syndrome – A systematic literature review

S. Zuberi, J. Sullivan, A. Deighton, 2022, Epilepsy & Behavior.

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

J. Mendell, A. Pickard, W. Arnold, 2018, Pediatric pulmonology.

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)

P. Cyr, D. Malone, T. Wiesner, 2021, Journal of market access & health policy.

Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients

P. Cyr, D. Malone, R. Arjunji, 2019, Journal of market access & health policy.

Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1

J. Jansen, R. Arjunji, O. Dabbous, 2019, Advances in Therapy.

Potential use of an estrogen-glucocorticoid receptor chimera as a drug screen for tissue selective estrogenic activity.

C. McArdle, J. Tobias, C. Caunt, 2009, Bone.

PRO35 ECONOMIC BURDEN OF CARE IMPOSED ON PATIENTS WITH RETT SYNDROME: A SYSTEMATIC LITERATURE REVIEW

O. Dabbous, R. Arjunji, B. Maru, 2019, Value in Health.