A new consortium promoting access to gene therapy for ultra-rare diseases
A new article published in Nature Medicine presents a new initiative tackling the challenges posed by existing models of for-profit drug marketing for promoting access to gene therapies for ultra-rare diseases. In the case of extremely small patient populations, commercialization of such treatments is not economically fit for purpose and sponsors are often unwilling to continue their involvement.
OpenEdition vous propose de citer ce billet de la manière suivante :
bartosb (5 octobre 2023). A new consortium promoting access to gene therapy for ultra-rare diseases. ELSIBI. Consulté le 16 mars 2026 à l’adresse https://doi.org/10.58079/o6ev





