Axon/ Neuromuscular Junction
2
2017
5
SOD1 Antisense Oligonucleotide (ASO)
1/2/3
2021
4
Neuromuscular Junction
2
2021
9
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
UNC13A Antisense Oligonucleotide (ASO)
1
2027
5
Sigma‑1 receptor agonist
3
2029
3
Axon Degeneration
1/2
2028
3
Blood Brain Barrier
1
2027
1
CAPN2 Antisense Oligonucleotide (ASO)
1
2027
5
Neuromuscular Junction
2
2026
2