Rare Disease and Orphan Drugs Journal
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Editor-in-Chief: Daniel Scherman
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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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Exploring new therapeutics for Duchenne muscular dystrophy and related cardiomyopathy

Bin Li, ... Alex C. Y. Chang
Open Access Review DOI: 10.20517/rdodj.2024.44 2 Apr 2025
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Rare diseases clinical trials toolbox - public resources and main considerations to set up a clinical trial on medicinal products for humans in Europe

Marta del ÁlamoMarta del Álamo, ... Christine Kubiak
Open Access Original Article DOI: 10.20517/rdodj.2024.49 20 Mar 2025
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The novel SCD MED ALERT mHealth app: empowering patients with sickle cell disease

Simone Villaboni, ... Lucia De Franceschi
Open Access Original Article DOI: 10.20517/rdodj.2024.31 17 Mar 2025
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Lymphedema and erysipelas in patients with classic Fabry disease: a retrospective case series

Dominique P. Germain, ... Lynda Barache
Open Access Original Article DOI: 10.20517/rdodj.2024.61 11 Mar 2025
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The necessity for skeletal muscle contractile assays to assess treatment efficacy in DMD

Claire Yuan, ... Robert W. Grange
Open Access Review DOI: 10.20517/rdodj.2024.34 4 Mar 2025
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Sustainable approaches for drug repurposing in rare diseases: recommendations from the IRDiRC Task Force

Galliano Zanello, ... Virginie Hivert
Open Access Perspective DOI: 10.20517/rdodj.2023.04 24 Apr 2023
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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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Overcoming challenges in sustaining newborn screening in low-middle-income countries: the Philippine newborn screening system

Carmencita D. PadillaCarmencita D. Padilla, ... Bradford L. TherrellBradford L. Therrell
Open Access Perspective DOI: 10.20517/rdodj.2023.38 10 Dec 2023
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Themed Collections
rare disease
orphan drug
Topic: Treatment in Autism Spectrum Disorder
Topic: Advancements in Diagnosis and Treatment of Fabry Disease 1.0
Topic: Newborn Screening I - Real World Applications and Technologies
Volume 4, Issue 1（ in progress）
Volume 3, Issue 4
Volume 3, Issue 3
Volume 3, Issue 2
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
Special Interview with Prof. Phillip D.K. Lee
Exploring the Frontiers of Pediatric Endocrinology and Scientific Publishing
The 2nd RDODJ Webinar
Familial Hypercholesterolemia
Special Interview with Dr. Randi J. Hagerman
During the interview, Dr. Hagerman elaborated on the key points of her article, current research, and prospects within the field of Fragile X Syndrome, and offered suggestions for the future development of the journal.
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

Sustainable approaches for drug repurposing in rare diseases: recommendations from the IRDiRC Task Force
Galliano Zanello, ... Virginie Hivert
Open Access | Perspective | 24 Apr 2023
Views: | Downloads:
Download PDF
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
Overcoming challenges in sustaining newborn screening in low-middle-income countries: the Philippine newborn screening system
Carmencita D. PadillaCarmencita D. Padilla, ... Bradford L. TherrellBradford L. Therrell
Open Access | Perspective | 10 Dec 2023
Views: | Downloads:
Download PDF
Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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