Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation

​ Conditions: Dentatorubral-Pallidoluysian Atrophy

Interventions: Drug: nL-ATN1-001

Sponsors: n-Lorem Foundation; Dell Children’s Medical Center of Central Texas

Not yet recruiting Conditions: Dentatorubral-Pallidoluysian Atrophy
Interventions: Drug: nL-ATN1-001
Sponsors: n-Lorem Foundation; Dell Children’s Medical Center of Central Texas
Not yet recruiting