Special Issue "Development of Medicines for Rare Pediatric Diseases II"

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmaceutical Technology".

Deadline for manuscript submissions: 15 December 2023 | Viewed by 112

Special Issue Editors

Department of Pharmacy, Università degli Studi di Genova, Genoa, Italy
Interests: retinoids; drug delivery systems; polymeric micelles; liposomes; passive targeting; active targeting; poorly soluble drugs; formulation strategies; cancer; neuroblastoma; melanoma; tumor mouse models; oncology; apoptosis
Special Issues, Collections and Topics in MDPI journals
Stem Cell Laboratory and Cell Therapy Center, IRCCS Istituto Giannina Gaslini, via G. Gaslini 5, 16147 Genova, Italy
Interests: cell biology; extracellular vesicles; tumors
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

It is estimated that up to 7000 rare diseases exist, affecting between 6% and 8% of the global population and >30 million people in the European Union. Symptoms often occur early at birth or during childhood and may progressively increase, becoming chronic or relapsing, leading to life-threatening conditions. In fact, 75% of rare diseases are known to affect children, and about 3 out of 10 of these children die before reaching 5 years of age. Therefore, incentivizing medicine development is crucial for this subset of the population, who is disproportionately affected by rare diseases. Furthermore, only 5% of rare diseases have effective treatment options, which leaves 95% of rare diseases without any approved medicines. Since both the Orphan Regulation, first, and the Paediatric Regulation, later, came into force, the development of medicinal products suitable for pediatrics has consistently increased, but much more needs to be done. To date, our medical and scientific knowledge on rare diseases remains limited, and the absence of approved treatment options has led physicians and pharmacists to the harmful off-label use of medicines. This Special Issue is addressed to all researchers and partners involved in the development of pediatric medicines, with the aim of collecting opinions from industry, regulators, academia, and hospitals, thus contributing to providing solutions to the unmet pediatric needs.

We welcome original research, reviews, opinion papers, editorials, or short communications on the following topics:

  • Pediatric formulations;
  • Drug-delivery design in pediatrics;
  • Orphan drugs;
  • Rare-disease treatment;
  • The pharmacology and toxicology of drugs and excipients in pediatrics;
  • Translational research in pediatrics;
  • Pediatric drug targeting;
  • Extemporaneous pediatric preparations compounded in hospital pharmacies.

Dr. Guendalina Zuccari
Dr. Danilo Marimpietri
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceuticals is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.


  • pediatric formulations
  • drug-delivery design in pediatrics
  • orphan drugs
  • rare-disease treatment
  • the pharmacology and toxicology of drugs and excipients in pediatrics
  • translational research in pediatrics
  • pediatric drug targeting
  • extemporaneous pediatric preparations compounded in hospital pharmacies

Published Papers

This special issue is now open for submission.
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