Diseases (the MPS Society), Registered Charity in England and Wales No 1143472. Rare Disease Research Partners social objectives are to reinvest any surplus to support the mission of the MPS Society to transform the lives of patients through specialist knowledge, support, advocacy and research....
Such studies are the first steps in rare disease research powered by empowered patients by solving the zebra rare disease puzzle through social media.doi:10.1002/cncr.34765Gilberto MorganVivek SubbiahCancer.
Investment in disease areas with blockbuster potential, such as Alzheimer’s and obesity, is overshadowing the needs of rare disease research. To address this imbalance, regulatory bodies must play a pivotal role in encouraging a more diversified investment landscape by offering incentives for R&D in...
"Rare disease research is a priority in Canada. With advances in genetics and genomics, we have the ability to ensure rare diseases are diagnosed and managed more efficiently and effectively, and we aspire to see this integrated in Canadian health care delivery," said Dr. Chr...
Future research focused on the implementation of such care pathways is a priority.Background In Europe, a rare disease (RD) is defined as a condition with less than five affected persons per 10,000 [1]. It is estimated that 300,000 people in Ireland are living with one of over 6000 ...
Orphan diseaseResearch collaborationorphan drugsorphan productsOffice of Rare diseases researchlt;pgt;lt;stronggt;Introductionlt;/stronggt;: Rare diseases present unique challenges to meet the numerous and varied needs of the rare diseases community and it is required to identify and address these ...
Sobi is dedicated to developing and delivering innovative therapies to improve the lives of rare disease patients.
Protheragen specializes in providing a range of preclinical research services for rare disease research, addressing many of the challenges encountered in drug discovery and development. Its end-to-end rare disease therapy development solutions cover a full package of the...
MedGenome Services CEO Dr. Vedam Ramprasad, said, "Few people realize that India and South Asia have the world's largest population of people affected by rare and inherited disease. Given the high unmet need of these patients and families, we are dedicated to expanding support and opportunities...
Our specialized testing provides actionable insights in genetic and rare diseases, helping to improve patient care and pharmaceutical drug research and development. Collaborate with Quest Expansive and deep testing capabilities Quest Diagnostics is at the forefront of genetics and rare disease diagnostics, ...