Abstract
I am grateful for this opportunity to serve as the new Editor-in-Chief of Therapeutic Advances in Rare Disease. No one could have known when this journal was launched in February 2020 that we would soon be plunged into a global pandemic the likes of which had not been seen for a century. It was a challenging time at all levels, personal, family, community, national, and global. It has had a significant impact on the lives of many individuals with rare disease and their ability to gain access to services and treatment. The ability to conduct research studies was also slowed, raising concerns among patients and caregivers as to how this would impact ongoing studies related to the advancement of therapeutics for rare diseases. Note that I said it “slowed” not stopped. The research community was driven to think “outside of the box” to enable them to conduct their studies using modified and novel approaches. I would like to take a moment to applaud the rare diseases research community for their commitment, tenacity, and ingenuity in keeping science moving forward during these challenging times.
Although most journals target a specific disease, organ, system, or age group, Therapeutic Advances in Rare Disease has the potential to touch on over 7000 diseases, that can affect multiple organs, systems, or age groups. In rare disease research, it is important that we are open to identifying commonalities across disorders, whether it is a common pathway, an innovative research design or a treatment that has the potential to benefit multiple disorders. Therapeutic Advances in Rare Disease is a peer-reviewed, gold open-access journal that provides a unique opportunity for our readers to be exposed to studies that may be within their area of research, as well as areas with which may be less familiar but may provide important insights that can translate into their own fields.
I am excited to be working with a team of world-renowned Associate Editors and a highly regarded international Editorial Board, who bring a wealth of experience and knowledge to guarantee the highest standards of publication. The aim of Therapeutic Advances in Rare Disease is to publish high-quality editorials, reviews, and rigorous original research articles relating to the innovative development of treatments for rare disease patients. Our goal is to be the first choice for prompt publication of results from rigorous, well-designed clinical studies. In this context, we want to encourage the reporting of preliminary data as well as negative clinical studies. We also publish observational studies and basic research with clinical relevance. A personal goal is to also provide opportunities for our next generation of rising stars in rare disease research.
The journal is committed to supporting open science initiatives and strengthening the patient voice in academic journals. We will do so by working with patient experts and patient groups to further validate research and publishing plain language summaries alongside articles to help increase the understanding of papers for non-specialists.
I am grateful for the continued support of you our readers, as well as to our authors and reviewers who supported us through the challenges of the past year. It is an exciting and hopeful time to be working in the field of rare disease research and I very much look forward to submissions that will help drive the field of rare disease therapeutics forward.