Several factors contribute to the difficulty of accurately identifying and counting rare diseases. On one hand, the definition of what constitutes a rare disease differs between different countries around the world, ranging from five to 76 affected individuals per 100,000 people in the general population.In...
Οrphan medicinal products (OMPs) are pharmaceuticals designed for the treatment of rare diseases, a family of more than 6000 medical conditions, each of which affects a small to ultra-small population of patients – typically less than 1 in 2000 people. The June 2009 European Council Recommendation...
Rare diseases represent a highly heterogeneous group of disorders with high phenotypic and genotypic diversity within individual conditions. Due to the small numbers of people affected, there are unique challenges in understanding rare diseases and drug development for these conditions, including patient identification and recruitment,...
Patients suffering from rare diseases deserve the same quality of treatment as other patients within the European Union. Given the small numbers of patients affected by rare diseases, the pharmaceutical industry has been reluctant in the past to invest in the research and development of medicinal...
WASHINGTON, May 19, 2020 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®) has announced new awards through its Rare Disease Research Grant Program, which provides funding to qualified researchers for translational or clinical studies related to the development of new diagnostics or treatments for...
In the run-up to Rare Disease Day 2019, David H. Crean, PhD, Managing Director for Objective Capital Partners, reviews the investment and deal activity for orphan drugs in 2018 and delves into future forecasts for the rare disease space.With drugs currently available for only about...
