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The complexity of funding rare disease research: an IRDiRC assessment of the landscape
Open Access Review 8 Sep 2024
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Non-viral gene therapy for neuromuscular diseases including Duchenne muscular dystrophy using nanovesicles derived from human cells
Open Access Review 1 Sep 2024
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Exploratory survey study on adjunctive use of medical cannabis for Gaucher disease
Open Access Original Article 22 Aug 2024
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Patient organizations: advocating for timely newborn screening & improved quality of life
Open Access Perspective 14 Aug 2024
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Framing the European Rare Diseases field through a structured movement of patient organisations
Open Access Perspective 30 Jul 2024
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Fabry nephropathy: a treatable cause of chronic kidney disease
Open Access Review 10 Jul 2024
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Gene therapy for Dravet syndrome: promises and impact on disease trigger and secondary modifications
Open Access Review 8 Jul 2024
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Incorporating a new disease in the newborn screening programs in Europe: the spinal muscular atrophy case study
Open Access Opinion 1 Jul 2024
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Longitudinal biomarker evaluation in Fabry disease patients receiving lentivirus-mediated gene therapy
Open Access Original Article 26 Jun 2024
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The European joint programme on rare diseases: building the rare diseases research ecosystem
Open Access Perspective 23 Jun 2024
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Newborn screening in Mexico and Latin America: present and future
Open Access Perspective 23 Jun 2024
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