The last day of February marks Rare Disease Day, an initiative that helps increase education, discussion, and improve access to treatment options. Orphanet Journal of Rare Diseases has rounded up a few interesting facts and research about rare diseases. Find out how much you know, and share your results with us @OJrarediseases and use the hash tag #RareDiseaseDay.
Orphanet Journal of Rare Diseases is proud to present this piece, authored by the Rare Diseases Team within the U.S. Food & Drug Administration’s (FDA) Division of Rare Diseases and Medical Genetics (Center for Drug Evaluation and Research, Office of New Drugs). The Team takes us through their productive efforts in approving new and/or repurposed treatments, for patients with rare diseases, and looks ahead to future efforts.
In this blog, Dr. Anne R. Pariser (Director of the Office of Rare Diseases Research, at the NIH’s National Center for Advancing Translational Sciences) discusses the milestones reached and benefits achieved for patients of both rare and common diseases, via rare disease research.
Orphanet Journal of Rare Diseases is proud to present this piece, a runner-up entry in Findacure and Medics4RareDiseases’ “Student Voice” contest, which explores some of the changes which need to be made in healthcare, in order to improve the experiences for patients with rare diseases.
**Please note: due to space constraints, this “Student Voice” blog appears in condensed form (the full-length version which was submitted for the contest, can be found here).
Patients with LAM (lymphangioleiomyomatosis) or other rare lung diseases can lose their exercise capacity because of dyspnea and decreased lung function. Effective treatments for rare diseases are often extremely limited. Yoga rehabilitation effectively improves exercise capacity for LAM patients, according to an article published in Orphanet Journal of Rare Diseases.
Orphanet Journal of Rare Diseases is proud to present this piece, a runner-up entry in Findacure and Medics4RareDiseases’ “Student Voice” contest, which explores some of the changes which need to be made in healthcare, in order to improve the experiences for patients with rare diseases.
**Please note: due to space constraints, this “Student Voice” blog appears in condensed form (the full-length version which was submitted for the contest, can be found here).
Orphanet Journal of Rare Diseases is proud to present this piece, a runner-up entry in Findacure and Medics4RareDiseases’ “Student Voice” contest, which explores some of the changes which need to be made in healthcare, in order to improve the experiences for patients with rare diseases.
**Please note: due to space constraints, this “Student Voice” blog appears in condensed form (the full-length version which was submitted for the contest, can be found here).
In a blog for Ovarian Cancer Awareness Month, Christina Uwins and Simon Butler-Manuel discuss the MIRRORS study registered at the ISRCTN registry.