Axon/ Neuromuscular Junction
2
2017
5
SOD1 Antisense Oligonucleotide (ASO)
1/2/3
2021
4
Neuromuscular Junction
2
2021
9
C9orf72 Antisense Oligonucleotide (ASO)
1
2022
4
C9orf72 Antisense Oligonucleotide (ASO)
1/2
2023
2
Blood Brain Barrier
1
2022
1
FUS Antisense Oligonucleotide (ASO)
3
2024
1
Ataxin 2 Antisense Oligonucleotide (ASO)
1
2026
1
SOD1 Antisense Oligonucleotide (ASO)
3
2027
2
STATHMIN-2 Antisense Oligonucleotide (ASO)
1
2025
4
Excitotoxicity
Open-label, pilot
2026
1
Neuromuscular Junction
2
2026
2