To the Editor — Rare and ultra-rare diseases affect millions of people worldwide. When treatments exist, they are usually directed at the symptomatic level rather than at the mechanistic basis of the disease. However, because many rare disorders stem from single-gene variations, they may be addressable using rapidly maturing genetic technologies, such as antisense oligonucleotides, small interfering RNAs, gene therapy and gene editing; indeed, a Comment in your June issue highlighted a not-for-profit initiative to address ultra-rare diseases using disease-modifying antisense oligonucleotides1. Although gene-based therapy for neurogenetic disorders remains in its infancy (with existing questions related to safety and efficacy), it may offer therapeutic opportunities for conditions that currently have few or no existing treatment options. Against this background, the US National Institute of Neurological Disorders and Stroke (NINDS) is launching a new program — the Ultra-rare Gene-based Therapy (URGenT) network — that will support the development of gene-based treatments for rare and ultra-rare diseases. The expectation is that such tailored treatments will result in improved patient outcomes for these diseases.
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The authors declare no competing interests.