Ultra-rare disease diagnosis remains an uphill battle
“In the vast majority of rare disorders, there are sufficient numbers of patients to test treatments. For example, in a trial you can give one group of patients an active medicine and a second group a placebo – a chemically inactive substance – to see if there is a significant difference. This can be hard in the case of ultra-rare diseases because there are so few patients.”
VASCERN covers many ultra-rare diseases, read more about the specific challenges encountered by these diseases here : https://www.raconteur.net/healthcare/ultra-rare-disease ... See more
