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Rare Disease and Orphan Drugs Journal
Open Access  ISSN: 2771-2893 (Online)   Editor-in-Chief: Daniel Scherman
Indexing: Dimensions, Lens,
MPT: 96 days
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Rare Disease and Orphan Drugs Journal

Open Access
ISSN: 2771-2893 (Online)
Editor-in-Chief: Daniel Scherman
Indexing: Dimensions, Lens,
MPT:  96 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

Bumetanide to treat Autism Spectrum Disorders: are complex administrative regulations fit to treat heterogeneous disorders?

Overcoming challenges in sustaining newborn screening in low-middle-income countries: the Philippine Newborn Screening System

The International Rare Disease Research Consortium (IRDiRC): making rare disease research efforts more efficient and collaborative, around the world

Articles

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Open Access Review

The Australian landscape of newborn screening in the genomics era

DOI: 10.20517/rdodj.2023.30
Charli Ji Charli Ji, Michelle A Farrar Michelle A Farrar, Sarah Norris Sarah NorrisSarah Norris, Kaustuv Bhattacharya Kaustuv Bhattacharya, Bruce Bennetts Bruce Bennetts, Ainsley J Newson Ainsley J Newson, Louise Healy Louise Healy, Nicole Millis Nicole Millis, Didu S Kariyawasam Didu S KariyawasamDidu S Kariyawasam
Available online: 23 Nov 2023
Open Access Review

Pharmacovigilance of gene therapy medicinal products

DOI: 10.20517/rdodj.2023.19
Nadine Petitpain Nadine PetitpainNadine Petitpain, Marie Lauren Antoine Marie Lauren Antoine, Mathilde Beurrier Mathilde Beurrier, Joelle Micallef Joelle MicallefJoelle Micallef, Audrey Fresse Audrey FresseAudrey Fresse
Available online: 22 Nov 2023
Open Access Review

Rare diseases with temporomandibular joint manifestations: a systematic review

DOI: 10.20517/rdodj.2023.32
Mayank Shrivastava Mayank Shrivastava, Gabriel Tian Gabriel Tian, Liang Ye Liang Ye
Available online: 20 Nov 2023
Open Access Original Article

2017-2023: state of the art of gene therapies in rare diseases in Europe: the dynamics of clinical R&D, new approved treatments and expected therapies in the pipelines

DOI: 10.20517/rdodj.2023.29
Tristan Gicquel Tristan Gicquel, Lucas Cortial Lucas Cortial, Karyna Lutsyk Karyna Lutsyk, Sylvain Forget Sylvain Forget, Serge Braun Serge Braun, Pierre-Olivier Boyer Pierre-Olivier Boyer, Vincent Laugel Vincent Laugel, Olivier Blin Olivier BlinOlivier Blin
Available online: 2 Nov 2023
Open Access Review

Federated learning for rare disease detection: a survey

DOI: 10.20517/rdodj.2023.16
Jiaqi Wang Jiaqi WangJiaqi Wang, Fenglong Ma Fenglong MaFenglong Ma
Available online: 9 Oct 2023
Open Access Editorial

Enzyme replacement therapy: current challenges and drug delivery prospects via extracellular vesicles

DOI: 10.20517/rdodj.2022.09
Amanda K. A. Silva Amanda K. A. SilvaAmanda K. A. Silva, Corinne Sagné Corinne SagnéCorinne Sagné, Florence Gazeau Florence GazeauFlorence Gazeau, Ibane Abasolo Ibane AbasoloIbane Abasolo
Available online: 3 Jul 2022
Open Access Opinion

Enhancing the value of clinical networks for rare diseases

DOI: 10.20517/rdodj.2022.01
Matthew Bolz-Johnson Matthew Bolz-Johnson, Louise Clément Louise Clément, William Gahl William GahlWilliam Gahl, Carmencita Padilla Carmencita Padilla, Yukiko Nishumura Yukiko Nishumura, Rachel Yang Rachel Yang, Lisa Sarfaty Lisa Sarfaty, Nicoline Hoogerbrugge Nicoline Hoogerbrugge, ... Thomas Kenny Thomas KennyThomas Kenny
Available online: 21 Apr 2022
Open Access Review

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

DOI: 10.20517/rdodj.2022.04
Thomas Kenny Thomas Kenny, Kathleen Bogart Kathleen Bogart, Albert Freedman Albert Freedman, Claire Garthwaite Claire Garthwaite, Susie M. D. Henley Susie M. D. Henley, Matt Bolz-Johnson Matt Bolz-Johnson, Shehla Mohammed Shehla Mohammed, Jill Walton Jill Walton, ... Deborah Woodman Deborah Woodman
Available online: 7 Apr 2022

Topic: Research Status of Spinal Muscular Atrophy

Dr. Marla Weetall; Dr. Nikolai Naryshkin

Submission Deadline: 31 Mar 2024

Published articles: 0

Topic: Advancements in Diagnosis and Treatment of Fabry Disease 2.0

Prof. Guillem Pintos-Morell; Prof. Derralynn Hughes

Submission Deadline: 31 Mar 2024

Published articles: 0

Data
219
Authors
197
Reviewers
43
Articles
55
On Board Editors
2021
Published Since
10
Special Issues
40,406
Article Views
19,870
Article Downloads
For Reviewers

Peer Review Guidelines
For Readers

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Themed Collections
rare disease
orphan drug
Volume 2, Issue 4 (in progress)
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

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

Bumetanide to treat Autism Spectrum Disorders: are complex administrative regulations fit to treat heterogeneous disorders?

Overcoming challenges in sustaining newborn screening in low-middle-income countries: the Philippine Newborn Screening System

The International Rare Disease Research Consortium (IRDiRC): making rare disease research efforts more efficient and collaborative, around the world

Open Access Editorial
Enzyme replacement therapy: current challenges and drug delivery prospects via extracellular vesicles
DOI: 10.20517/rdodj.2022.09
Amanda K. A. SilvaAmanda K. A. Silva, ... Ibane AbasoloIbane Abasolo
Available online: 3 Jul 2022
Open Access Opinion
Enhancing the value of clinical networks for rare diseases
DOI: 10.20517/rdodj.2022.01
Matthew Bolz-Johnson, ... Thomas KennyThomas Kenny
Available online: 21 Apr 2022
Open Access Review
The importance of psychological support for parents and caregivers of children with a rare disease at diagnosis
DOI: 10.20517/rdodj.2022.04
Thomas Kenny, ... Deborah Woodman
Available online: 7 Apr 2022

Topic: Research Status of Spinal Muscular Atrophy

Dr. Marla Weetall; Dr. Nikolai Naryshkin

Submission Deadline: 31 Mar 2024

Published articles: 0

Topic: Advancements in Diagnosis and Treatment of Fabry Disease 2.0

Prof. Guillem Pintos-Morell; Prof. Derralynn Hughes

Submission Deadline: 31 Mar 2024

Published articles: 0

Data
219
Authors
197
Reviewers
43
Articles
55
On Board Editors
2021
Published Since
10
Special Issues
40,406
Article Views
19,870
Article Downloads
Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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