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Review of methods for estimating the prevalence of rare diseases

Open Access Review DOI: 10.20517/rdodj.2023.39 17 Feb 2024
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Investigating Fabry disease - some lessons learned

Open Access Review DOI: 10.20517/rdodj.2023.50 22 Jan 2024
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Management of sleep problems in people with autism: an updated review

Open Access Review DOI: 10.20517/rdodj.2023.36 15 Jan 2024
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Light chain (AL) amyloidosis following gastrointestinal symptoms that involve multiple organs: a case report

Open Access Case Report DOI: 10.20517/rdodj.2023.45 2 Jan 2024
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Bumetanide to treat autism spectrum disorders: are complex administrative regulations fit to treat heterogeneous disorders?

Open Access Original Article DOI: 10.20517/rdodj.2023.22 2 Jan 2024
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The international rare disease research consortium (IRDiRC): making rare disease research efforts more efficient and collaborative around the world

Open Access Review DOI: 10.20517/rdodj.2023.23 13 Dec 2023
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Overcoming challenges in sustaining newborn screening in low-middle-income countries: the Philippine newborn screening system

Open Access Perspective DOI: 10.20517/rdodj.2023.38 10 Dec 2023
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The Australian landscape of newborn screening in the genomics era

Open Access Review DOI: 10.20517/rdodj.2023.30 23 Nov 2023
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Pharmacovigilance of gene therapy medicinal products

Open Access Review DOI: 10.20517/rdodj.2023.19 22 Nov 2023
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Rare diseases with temporomandibular joint manifestations: a systematic review

Open Access Review DOI: 10.20517/rdodj.2023.32 20 Nov 2023
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Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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