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Aartsma-Rus A, Corey DR. The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy. Nucleic acid therapeutics 2020;30:67-70   Introduction…

Vat LE, Finlay T, Jan Schuitmaker-Warnaar T, et al. Evaluating the "return on patient engagement initiatives" in medicines research and…

Nguengang Wakap S, Lambert DM, Olry A, et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet…

Khodyakov D, Grant S, Denger B, et al. Practical Considerations in Using Online Modified-Delphi Approaches to Engage Patients and Other…

Verhaart IEC, ‘t Hoen PAC, Roos M, Vroom E. Meeting on data sharing for Duchenne 21–22 March 2019 Amsterdam, the…

Verhaart IEC, Johnson A, Thakrar S, et al. Muscle biopsies in clinical trials for Duchenne muscular dystrophy – Patients’ and…

Courbier S, Dimond R, Bros-Facer V. Share and protect our health data: an evidence based approach to rare disease patients'…

Lang SM, Alsaied T, Moore RA, Rattan M, Ryan TD, Taylor MD. Conservative gadolinium administration to patients with Duchenne muscular…

Haberkamp M, Moseley J, Athanasiou D, et al. European regulators' views on a wearable-derived performance measurement of ambulation for Duchenne…

Atehortua SC, Lugo LH, Ceballos M, et al. Cost-Effectiveness Analysis of Diagnosis of Duchenne/Becker Muscular Dystrophy in Colombia. Value in…