Rare Disease and Orphan Drugs Journal
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Editor-in-Chief: Daniel Scherman
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Submission to first decision: 72 days
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Rare Disease and Orphan Drugs Journal
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Editor-in-Chief: Daniel Scherman
Submission to first decision: 72 days
Home Articles Special Issues Volumes Webinars Videos

Rare Disease and Orphan Drugs Journal

The importance of psychological support for parents and caregivers of children with a rare disease at diagnosis

The dynamic and urgent path of rare disease and orphan drug research

Biomarkers in Anderson-Fabry disease: what should we use in the clinical practice?

Analysis of genomics implementation in Newborn Screening for Inherited Metabolic Disorders: an IRDiRC initiative

Articles

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Gastrointestinal involvement in Fabry disease

Juan Manuel PoliteiJuan Manuel Politei, Benjamin Solar
Open Access Review DOI: 10.20517/rdodj.2023.46 28 Mar 2024
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Cerebrovascular disorders and Fabry disease

David Moreno-MartínezDavid Moreno-Martínez, ... Ricardo ReisinRicardo Reisin
Open Access Review DOI: 10.20517/rdodj.2023.51 24 Mar 2024
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Paradox of possibilities: the rare landscape in the Asia Pacific Region

Ritu JainRitu Jain, ... Kin Ping Tsang
Open Access Perspective DOI: 10.20517/rdodj.2023.42 28 Feb 2024
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Newborn screening in South Africa: the past, present, and plans for the future

Helen L. Malherbe, ... Barend C. Vorster
Open Access Commentary DOI: 10.20517/rdodj.2023.49 28 Feb 2024
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ERCAL, a regional initiative for rare diseases in Latin America and the Caribbean

Claudia Gonzaga-JaureguiClaudia Gonzaga-Jauregui, ... Stephen C. Groft
Open Access Perspective DOI: 10.20517/rdodj.2023.48 26 Feb 2024
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Connecting academia and industry for innovative drug repurposing in rare diseases: it is worth a try

Christine Fetro
Open Access Perspective DOI: 10.20517/rdodj.2023.06 9 Apr 2023
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Enzyme replacement therapy: current challenges and drug delivery prospects via extracellular vesicles

Amanda K. A. SilvaAmanda K. A. Silva, ... Ibane AbasoloIbane Abasolo
Open Access Editorial DOI: 10.20517/rdodj.2022.09 3 Jul 2022
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Enhancing the value of clinical networks for rare diseases

Matthew Bolz-Johnson, ... Thomas KennyThomas Kenny
Open Access Opinion DOI: 10.20517/rdodj.2022.01 21 Apr 2022
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Topic: Sickle Cell Disease(SCD): Progress in Research and Insights

Dr. Marina Cavazzana; Dr. Thiago Trovati MacIel

Submission Deadline: 31 Aug 2024

Published articles: 0

Topic: Research Status of Spinal Muscular Atrophy

Dr. Marla Weetall; Dr. Nikolai Naryshkin

Submission Deadline: 30 Jun 2024

Published articles: 0

Data
282
Authors
256
Reviewers
55
Articles
55
On Board Editors
2021
Published Since
13
Special Issues
50,742
Article Views
22,287
Article Downloads
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Themed Collections
rare disease
orphan drug
Volume 3, Issue 2 (in progress)
Volume 3, Issue 1
Volume 2, Issue 4
Volume 2, Issue 3
Volume 2, Issue 2
Volume 2, Issue 1
Volume 1, Issue 4
Volume 1, Issue 3
Volume 1, Issue 2
Volume 1, Issue 1
Rare Disease and Orphan Drugs Journal Supports 2024 Global Rare Diseases Research Symposium
Rare Disease and Orphan Drugs Journal is delighted to announce support for the 2024 Global Rare Disease Research Symposium
Series special issues on Newborn Screening

The importance of psychological support for parents and caregivers of children with a rare disease at diagnosis

The dynamic and urgent path of rare disease and orphan drug research

Biomarkers in Anderson-Fabry disease: what should we use in the clinical practice?

Analysis of genomics implementation in Newborn Screening for Inherited Metabolic Disorders: an IRDiRC initiative

Connecting academia and industry for innovative drug repurposing in rare diseases: it is worth a try
Christine Fetro
Open Access | Perspective | 9 Apr 2023
Views: | Downloads:
Download PDF
Enzyme replacement therapy: current challenges and drug delivery prospects via extracellular vesicles
Amanda K. A. SilvaAmanda K. A. Silva, ... Ibane AbasoloIbane Abasolo
Open Access | Editorial | 3 Jul 2022
Views: | Downloads:
Download PDF
Enhancing the value of clinical networks for rare diseases
Matthew Bolz-Johnson, ... Thomas KennyThomas Kenny
Open Access | Opinion | 21 Apr 2022
Views: | Downloads:
Download PDF

Topic: Sickle Cell Disease(SCD): Progress in Research and Insights

Dr. Marina Cavazzana; Dr. Thiago Trovati MacIel

Submission Deadline: 31 Aug 2024

Published articles: 0

Topic: Research Status of Spinal Muscular Atrophy

Dr. Marla Weetall; Dr. Nikolai Naryshkin

Submission Deadline: 30 Jun 2024

Published articles: 0

Data
282
Authors
256
Reviewers
55
Articles
55
On Board Editors
2021
Published Since
13
Special Issues
50,742
Article Views
22,287
Article Downloads
Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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