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Therapeutic Peptides Expanded 2 intermediate

Risdiplam: Oligopeptide Research Reference

Risdiplam, a bioactive peptide compound with documented applications in biomedical research, pharmacological studies, and therapeutic development

By Encyclopeptide Editorial | 1 min read
therapeutic-peptides-expanded-2 peptide oligopeptide

Overview

Risdiplam is a bioactive peptide with well-characterized properties and therapeutic applications.

Structure

PropertyValue
NameRisdiplam
SequenceSmall molecule SMN2 splicing modifier (pyridazine derivative)
LengthSmall molecule
Molecular Weight~372 Da

| Category | Rare Disease / RNA Splicing Modifier |

Mechanism of Action

Modifies splicing of SMN2 pre-mRNA to increase exon 7 inclusion, raising functional SMN protein levels in motor neurons and peripheral tissues. Oral small molecule alternative to intrathecal nusinersen

References

  • Wikipept Peptide Database
  • Primary literature (see individual entries)

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