Realising the potential of gene therapies for rare and ultra-rare inherited diseases. Claire Booth et al. Hum Gene Ther 2023 8

From the abstract: "Rare and ultra-rare diseases have been central to the field of gene therapy since its earliest stage, and we are now witnessing more and more effective treatments entering the clinical realm for patients in need. However, despite promising results across a range of rare diseases, transformative gene therapies may not be available and accessible to patients for non-medical reasons. Traditional regulatory and commercialisation pathways to licensed products seem to be prohibitive for ultra-small patient populations. Here we highlight some of the challenges of delivering gene therapies in rare diseases and discuss innovative solutions being proposed by the gene therapy community." https://phgkb.cdc.gov/PHGKB/phgHome.action?query=Gene%20therapy&action=search&Mysubmit=Search