Three years ago our son Thijs was born. All appeared fine for the first couple of hours, but his situation quickly deteriorated and soon he was admitted to the IC. After several weeks, he was diagnosed with an extremely rare genetic disease: Microvillus Inclusion Disease (MID) where the small intestine is unable to absorb nutrients. The doctors explained that no cure exists and that lifelong treatment with total parenteral nutrition would be required. We were also told that there was no treatment this Disease.
But today, there is hope for a cure since the UMC Amsterdam hospital announced that it is going to research the option to cure MID patients.
In the study, our son Thijs is one of the patients for whom small mini-intestines are being grown from stem cells. With these so-called 'mini-guts,' the effects of the MYO5B gene (the gene that causes MID) will be mapped, and potential treatments will be tested. Ultimately, the research will explore whether there are possibilities to apply a new revolutionary treatment, the so-called CRISPR-Cas technique.
The research has just started and has received funding, but more money is needed to make this research successful.
