Additional rare diseases which remain ‘funding orphans’ include certain cancers such as acinar carcinoma, autoimmune pancreatitis, and various types of diseases characterized by cysts, both benign and malignant, among others. For these reasons, we provide the following websites that deal with funding...
Clinical research for rare disease: opportunities, challenges, and solutions. Mol Genet Metab 2009; 96(1):20-26.Griggs RC, Batshaw M, Dunkle M, et al. Clinical research for rare disease: opportunities, challenges, and solutions. Mol Genet Metab 2009 Jan; 96 (1): 20-6...
The term “undiagnosed disease” has emerged as a concept and category which helps properly guide the patient's management within the health system and reduce the time to obtain a correct diagnosis. This new series from Orphanet Journal of Rare Diseases will delve into the questions which need ...
Government Support: Various governments are investing in rare disease research and diagnostics, providing funding and regulatory support. Rising Healthcare Expenditure: Higher healthcare spending globally is enabling more comprehensive diagnostic services for rare diseases. Challenges and Opportunities Challenges...
The mapping of the human genome has unlocked new opportunities for studying rare diseases, enabling personalized medicine and targeted drug development. Multidisciplinary collaboration enhances the potential for breakthrough discoveries. Sustained funding from government and private sectors is crucial for ...
Rare disease (RD) management and orphan drug development in India face various hurdles regarding the implementation and adoption of comprehensive policies, lack of dedicated regulatory frameworks, and absence of epidemiological data. Current rare disease
This limited opportunities for advancements in rare disease treatment and care. Solution: We created a breakthrough technology, the Data Elements Matrix (DEM), to unlock and enable aggregation of sensitive patient data. The DEM is a novel data structure that completely preserves the privacy of ...
Rare disease (RD) management and orphan drug development in India face various hurdles regarding the implementation and adoption of comprehensive policies, lack of dedicated regulatory frameworks, and absence of epidemiological data. Current rare disease
The European Union Committee of Experts on Rare Diseases: three productive years at the service of the rare disease community The European Union Committee of Experts on Rare Diseases was entrusted with aiding the European Commission in a number of tasks, ranging from the monitorin... S Aymé,C...
RD require the combined efforts of health and social care professionals, politicians, managers and researchers to increase the availability of effective disease management tools to improve care and to extend both life expectancy and Health Related Quality of Life. 展开 ...