N-lorem Foundation

This study is testing a custom-made drug designed specifically for one person with a rare, inherited nerve disorder called Charcot-Marie-Tooth disease type 2D. The drug is an antisense oligonucleotide, which is a type of molecule that aims to target the underlying genetic cause o…

This study is creating and testing a personalized drug for a single child with a rare genetic disorder called NEDBA. The drug is designed to target the child's specific genetic mutation. Researchers will check if the treatment is safe and if it helps improve the child's movement,…

This is a study for a single child with Bainbridge-Ropers syndrome, a rare genetic disorder that causes severe developmental delays. Researchers are testing a custom-made genetic drug designed specifically for the child's unique gene variant. The main goals are to see if the trea…

This study is testing a personalized genetic drug designed for a single patient with a rare form of inherited retinal dystrophy. The goal is to see if the custom treatment is safe and if it can help slow or stop the progression of vision loss. Researchers will carefully monitor t…