Families Pursue Clinical Trials for Ultra Rare Diseases

Jaci's story is the latest example of patients pursuing custom-designed antisense oligonucleotide (ASO) therapies. These compounds lend themselves to personalization, as their chemistry is well understood and their production is relatively simple. As patients become increasingly savvy about raising money and awareness, researchers and regulators expect increased demand for personalized treatments with these compounds.There are at least five n-of-1 trials with ASOs. Jaci's is one