n-Lorem Foundation

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

Role: lead

Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

Role: lead

Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation Associated With Schuurs-Hoeijmakers Syndrome (SHMS)

Role: lead

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

Role: collaborator

Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

Role: lead

Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)

Role: lead

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

Role: lead

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation

Role: lead

Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)

Role: lead

Personalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2 Mutation Associated With Retinal Dystrophy

Role: lead

Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

Role: lead

Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

Role: lead

Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS

Role: lead

Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation

Role: lead

Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

Role: lead

Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

Role: collaborator