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

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Long-term treatment with insulin-like growth factor-1 in Phelan-McDermid syndrome: a case report

Mara Parellada, ... María de los Desamparados Rodríguez
Open Access Case Report DOI: 10.20517/rdodj.2023.53 5 May 2024
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Biomarkers in anderson-Fabry disease: what should we use in the clinical practice?

Patrício AguiarPatrício Aguiar
Open Access Review DOI: 10.20517/rdodj.2023.56 24 Apr 2024
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Analysis of genomics implementation in newborn screening for inherited metabolic disorders: an IRDiRC initiative

Guillem Pintos-MorellGuillem Pintos-Morell, ... Francjan J. van SpronsenFrancjan J. van Spronsen
Open Access Opinion DOI: 10.20517/rdodj.2023.52 23 Apr 2024
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The inflammatory pathogenetic pathways of Fabry nephropathy

Sandro FeriozziSandro Feriozzi, Paula Rozenfeld
Open Access Review DOI: 10.20517/rdodj.2023.37 17 Apr 2024
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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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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: Prader-Willi Syndrome: Clinical, Genetic, and Endocrine Research

Maurizio Delvecchio, PhD

Submission Deadline: 15 Nov 2024

Published articles: 0

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

Data
298
Authors
264
Reviewers
59
Articles
55
On Board Editors
2021
Published Since
14
Special Issues
52,606
Article Views
22,945
Article Downloads
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rare disease
orphan drug
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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

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: Prader-Willi Syndrome: Clinical, Genetic, and Endocrine Research

Maurizio Delvecchio, PhD

Submission Deadline: 15 Nov 2024

Published articles: 0

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

Data
298
Authors
264
Reviewers
59
Articles
55
On Board Editors
2021
Published Since
14
Special Issues
52,606
Article Views
22,945
Article Downloads
Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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