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Facey KM, Rannanheimo P, Batchelor L, Borchardt M, de Cock J. Real-world evidence to support Payer/HTA decisions about highly innovative…

Smith J, Damm K, Hover G, Chien J. Lessons from an Experiential Approach to Patient Community Engagement in Rare Disease.…

Whichello C, Bywall KS, Mauer J, Stephen W, Cleemput I, Pinto CA, et al. An overview of critical decision-points in…

Srivastava P, Malhotra KP, Husain N, Malhotra HS, Kulshreshtha D, Anand A. Diagnosing Muscular Dystrophies: Comparison of Techniques and Their…

Dhillon S. Viltolarsen: First Approval. Drugs 2020;80:1027-1031   Abstract Viltolarsen (Viltepso((R)) in Japan) is a phosphorodiamidate morpholino antisense oligonucleotide being…

Desviat LR, Mallebrera CJ, Vallejo-Illarramendi A, Mayan MD, Nogales-Gadea G, Arechavala-Gomeza V. COST Actions: fostering collaborative research for rare diseases.…

Flores D, Ribate MP, Montolio M, Ramos FJ, Gomez M, Garcia CB. Quantifying the economic impact of caregiving for Duchenne…

Crossnohere NL, Fischer R, Crossley E, Vroom E, Bridges JF. The evolution of patient-focused drug development and Duchenne muscular dystrophy.…

Menon D, Stafinski T, Dunn A, Short H. Involving patients in reducing decision uncertainties around orphan and ultra-orphan drugs: a…

Heslop E, Csimma C, Straub V, et al. The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to…