Development of Medicines for Rare Pediatric Diseases
A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmaceutical Technology".
Deadline for manuscript submissions: closed (17 February 2023) | Viewed by 45887
Special Issue Editors
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
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, the 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 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.
Keywords
- 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
Benefits of Publishing in a Special Issue
- Ease of navigation: Grouping papers by topic helps scholars navigate broad scope journals more efficiently.
- Greater discoverability: Special Issues support the reach and impact of scientific research. Articles in Special Issues are more discoverable and cited more frequently.
- Expansion of research network: Special Issues facilitate connections among authors, fostering scientific collaborations.
- External promotion: Articles in Special Issues are often promoted through the journal's social media, increasing their visibility.
- e-Book format: Special Issues with more than 10 articles can be published as dedicated e-books, ensuring wide and rapid dissemination.
Further information on MDPI's Special Issue polices can be found here.