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Article / Letter to editor
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2021
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Leiden University Medical Center (LUMC)
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Medicine / Leiden University Medical Centre (LUMC)
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rare disease
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Aartsma-Rus, A.
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en
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Applying lessons learned from developing exon skipping for duchenne to developing individualized exon skipping therapy for patients with neurodegenerative diseases
Expanding the HPSE2 genotypic spectrum in Urofacial Syndrome
Orphan medicine incentives
The role of patient involvement when developing therapies
The Matchmaker Exchange: A Platform for Rare Disease Gene Discovery
The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia