The following resources can be used to stay informed about new treatments and research results related to a rare disease:PubMed
PubMed is a database of published research articles. To find published results, try searching PubMed using the name of the rare disease. To find results from a specific research study, use the lead researcher’s name and key words from the study as search terms. Results from completed studies may take several years to publish.ClinicalTrials.gov
Periodically checking in ClinicalTrials.gov
for clinical studies testing new treatments provides a way to stay informed about new therapies that may be available in the future. The most promising treatments will progress to Phase 3 studies.ClinicalTrials.gov
requires the results of certain types of studies to be entered into the Results Database. This is normally completed within one year of completion of the study. Results entered in the database can be found in the Study Results tab of the study.Patient and Disease Organizations
Patient and rare disease organizations may list summaries of research efforts supported by the organization or relevant to their disease(s).
Many organizations have a section of their website devoted to research news and updates. This may include a list recently published research papers; a summary of the findings; items from the popular press; and information about funded researchers, members of their scientific advisory board, or the pharmaceutical industry.