Ontology highlight
ABSTRACT:
SUBMITTER: Suarez-Herrera N
PROVIDER: S-EPMC11011354 | biostudies-literature | 2024 Mar
REPOSITORIES: biostudies-literature
Suárez-Herrera Nuria N Li Catherina H Z CHZ Leijsten Nico N Karjosukarso Dyah W DW Corradi Zelia Z Bukkems Femke F Duijkers Lonneke L Cremers Frans P M FPM Hoyng Carel B CB Garanto Alejandro A Collin Rob W J RWJ
Cells 20240329 7
Precision medicine is rapidly gaining recognition in the field of (ultra)rare conditions, where only a few individuals in the world are affected. Clinical trial design for a small number of patients is extremely challenging, and for this reason, the development of N-of-1 strategies is explored to accelerate customized therapy design for rare cases. A strong candidate for this approach is Stargardt disease (STGD1), an autosomal recessive macular degeneration characterized by high genetic and phen ...[more]