Novel Strategies in Gangliosidoses Therapy
A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmacology".
Deadline for manuscript submissions: 22 August 2024 | Viewed by 134
Special Issue Editor
2. Department of Pediatrics, University of Minnesota, 2450 Riverside Avenue, Minneapolis, MN 55454, USA
3. Department of Experimental and Clinical Pharmacology, College of Pharmacy, University of Minnesota, 420 Delaware St SE, MMC 446, Minneapolis, MN 55455, USA
4. Advanced Therapies Program, University of Minnesota (UMMC) and Fairview Hospitals, Minneapolis, MN 55454, USA
Interests: gangliosidosis diseases; enzyme replacement therapies; pharmacokinetics and pharmacodynamics of ERT; small-molecule therapies
Special Issue Information
Dear Colleagues,
The Orphan Drug Act (passed in the United States in 1983) and the European Rare Disease Program (adopted by the European Union Counsel in 1995) are examples of legislations that ushered in an era of drug development aimed at creating therapies for orphan diseases. Orphan diseases are rare diseases, for which the rarity limits market size, thereby limiting resources for drug development. The various rare disease legislations encourage rare disease therapy development by implementing a number of methods. These can include tax incentives and clinical research subsidies for rare disease drug development, lowering statistical burden for clinical trials involving small patient populations, and creating research enterprises at government level to help guide and enable rare disease drug development.
This Special Issue of Pharmaceuticals examines the therapy development landscape for gangliosidoses, a group of rare diseases that have sometimes been further defined as “ultra-rare”, due to the exceptionally small worldwide patient population. One might also say that gangliosidoses have been “ultra-orphaned”, not only by disease rarity, but also by challenges in treating the central nervous system pathology. The longstanding absence of gangliosidosis-targeted therapies finally ended in recent years, following the entry of several gene therapies and small-molecule therapies into clinical trials. Research momentum continues to increase with more therapies being researched and more clinical trials being planned.
This Pharmaceuticals journal invites researchers to submit reviews and original articles that will enhance our understanding of the natural history of the gangliosidoses, therapies for gangliosidoses (e.g., small molecules, gene therapies, enzyme replacement, and palliative care), the challenges in treating the central nervous system, and meaningful outcome measures for gangliosidosis clinical trials.
Dr. Jeanine Jarnes
Guest Editor
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
- gangliosidoses
- small molecules
- gene therapies
- enzyme replacement
- palliative care
