Individualized therapy development for rare diseases: individualized at every step of the way

Therapeutic Advances in Rare Disease, vol. 6, pp. 26330040251386869

Authors 3

1. Marlen C. Lauffer Aachen Dutch Center for RNA Therapeutics Department of Human Genetics

   Leiden University Medical Center

   Affiliation as printed

   Dutch Center for RNA Therapeutics, Department of Human Genetics, Leiden University Medical Center, The Netherlands

   N=1 Collaborative, 7 Carole Place, Somerville, MA 02143, USA

2. Timothy W. Yu

   Boston Children's Hospital

   Affiliation as printed

   Boston Children’s Hospital, Boston, MA, USA

   N=1 Collaborative, 7 Carole Place, Somerville, MA 02143, USA

3. Annemieke M Aartsma-Rus corresponding Aachen Dutch Center for RNA Therapeutics Department of Human Genetics

   Leiden University Medical Center

   Affiliation as printed

   Dutch Center for RNA Therapeutics, Department of Human Genetics, Leiden University Medical Center, Albinusdreef 2, 2333 ZA Leiden, The Netherlands, N=1 Collaborative, 7 Carole Place, Somerville, MA 02143, USA