MD is one of the most prevalent genetic diseases, yet very unknown. That’s problematic as it results in massive underdiagnosis. As a consequence, people with very serious and ultimately lethal, medical issues do not get the support they need. This includes cardiac and cognitive issues, gastrointestinal problems, muscle wasting, amongst others. As an inherited disease, multiple people in a family have the condition per definition, increasing the burden on families.

Our colleague Jorg van Gent has this disease running through his family, including both his daughter and partner. They have set-up a charity MD Europe to raise awareness and pro-actively accelerate drug development in The Netherlands and other European countries.
This cause meets the criteria of effective giving: (1) it’s a neglected issue, (2) it has scale, as the largest muscle disease, and (3) there is tech in development to get to a cure in the next five years.