Risdiplam (RG7916) (Synonyms: RG-7916, RO7034067) |
| Catalog No.GC30845 |
Risdiplam (RG7916) (RG7916) is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels.
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Cas No.: 1825352-65-5
Sample solution is provided at 25 µL, 10mM.
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Risdiplam is a small molecule SMN2 pre-mRNA splicing modifier that promotes the inclusion of exon 7 and production of full-length SMN2 mRNA, which can compensate for the loss of SMN1 [1-2].
Risdiplam (RG7916)(0-1µM) was active in vitro in SMA patient-derived fibroblasts and in motor neurons generated from induced pluripotent stem cells (iPSCs) derived from SMA type 1 patient fibroblasts, promoting the inclusion of exon 7, to generate full-length (FL) mRNA[1].
Risdiplam (RG7916) potently increases SMN protein in both brain and muscle tissues of transgenic mouse models of SMA[1]. In infants with type 1 spinal muscular atrophy, treatment with oral risdiplam(0.08-0.2mg/kg/day)led to an increased expression of functional SMN protein in the blood[3].
References:
[1]. Ratni H, Ebeling M,et,al. Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Aug 9;61(15):6501-6517. doi: 10.1021/acs.jmedchem.8b00741. Epub 2018 Jul 25. PMID: 30044619.
[2]. Singh RN, Ottesen EW, et,al. The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy. Neurosci Insights. 2020 Nov 23;15:2633105520973985. doi: 10.1177/2633105520973985. PMID: 33283185; PMCID: PMC7691903.
[3]. Baranello G, Darras BT, et,al. FIREFISH Working Group. Risdiplam in Type 1 Spinal Muscular Atrophy. N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24. PMID: 33626251.
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