FIELD: medicine; pharmaceuticals.
SUBSTANCE: invention refers to compounds of formula (I), as well as their pharmaceutically acceptable salts, pharmaceutical compositions containing them and their use as medicinal agents.
EFFECT: obtaining novel compounds of formula (I), suitable for preparing drugs for modulating splicing of SMN2 gene.
39 cl, 6 tbl, 51 ex
| Title | Year | Author | Number |
|---|---|---|---|
| COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY | 2014 |
|
RU2673542C2 |
| MOLECULAR GENETIC SYSTEM FOR DETECTION OF DELETION SMN1 EXON 7 SUITABLE FOR NEONATAL SCREENING | 2021 |
|
RU2796350C1 |
| COMPOSITIONS AND METHODS OF MODULATING SMN2 SPLICING IN SUBJECT | 2010 |
|
RU2566724C9 |
| PHYTOECDYSONES AND DERIVATIVES THEREOF FOR USE IN TREATING NEUROMUSCULAR DISEASES | 2020 |
|
RU2820936C2 |
| METHOD OF ASSESSING EFFECTIVENESS OF THERAPEUTIC CONSTRUCTS AIMED AT INCREASING LEVEL OF FULL-LENGTH SMN TRANSCRIPTS | 2022 |
|
RU2811909C1 |
| METHOD OF PREIMPLANTATION GENETIC DIAGNOSTICS OF TYPE 1 SPINAL MUSCULAR ATROPHY | 2017 |
|
RU2671156C1 |
| GENETIC CONSTRUCT ADAPTED TO DELIVER THE HUMAN SMN1 GENE WITH ADENO-ASSOCIATED VIRUS OF SEROTYPE 2 TO PROVIDE NEUROSPECIFIC EXPRESSION | 2022 |
|
RU2801848C1 |
| COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT | 2019 |
|
RU2793459C2 |
| COMPOSITIONS AND METHODS OF SML2 SPLICING MODULATION IN SUBJECT | 2010 |
|
RU2683772C2 |
| SYNTHESIS METHOD OF 2'-O-(2-METHOXYETHYL) THIOPHOSPHATE OLIGONUCLEOTIDE | 2019 |
|
RU2738093C1 |
