Submitted by Anonymous (not verified) on 2 February 2024 - 9:51
Orphan designation: 2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting PLP1 pre-mRNA Treatment of Pelizaeus-Merzbacher disease, 16/08/2023 Positive
Source:
Orphan designation: 2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting PLP1 pre-mRNA Treatment of Pelizaeus-Merzbacher disease, 16/08/2023 Positive