Detailed view for this entity.
“There are more than 7,000 rare diseases that affect more than 300 million people globally. When you add in the families whose lives are touched by being able to support their family members with a rare disease, we're looking at a billion people in the world.”
“Rare diseases are one of the largest unmet needs in healthare systems globally. These conditions are individually rare but they're collectively common. And when we look at the financial impact of these diseases, it's often measured in the trillions. So we know that untreated rare diseases are a major drag on health care systems efficiency and productivity globally.”
“But in addition, what we know from the history of rare disease research is that rare diseases are fertile ground for research and innovation in terms of scientific understanding that impacts broader populations. And so there is a long track record of innovations that were incubated in rare disease research settings that ended up ultimately having broad societal impact that was very positive across healthcare systems.”
“But actually more we are expanding diagnosis for example, more we have the the ability to collect the data and also have newborn screening etc. more we realize that there are more actually person living with a rare disease than we would have expected at the very beginning. So it helped the recognition as well. We have many um person living with rare disease that come to us and say my disease is not being recognized in my country. If the disease is not recognized in the country, they're not in enroll in the health system. So they're not counted, they're not visible, they're not counted, they don't have care.”