Active Pharmaceutical Ingredients

English Synonyms RG-7916 RO-7034067 2-(2,8-Dimethylimidazo[1,2-b]pyridazin-6-yl)-7-(4,7-diazaspiro[2.5]octan-7-yl)-4H-pyrido[1,2-a]pyrimidin-4-one

Catalog ID101710 CAS NO.1825352-65-5

Purity 95%

MDL NumberMFCD31657372

EINEC Molecular Formula C22H23N7O

Molecular Weight 401.46

Category	Content
Generic Name	Risdiplam
Brand Name	Evrysdi
Dosage Form	Oral powder for reconstitution (0.75 mg/mL)
Drug Class	mRNA splicing modifier
Mechanism of Action	Risdiplam is an mRNA splicing modifier that targets the SMN2 gene to increase the production of functional SMN protein, which is deficient in patients with spinal muscular atrophy (SMA).
Indications	Treatment of spinal muscular atrophy (SMA) in patients aged 2 months and older.
Side Effects	Common side effects include fever, lung infection, vomiting, diarrhea, constipation, rash, and cold symptoms.
Storage	Store in a refrigerator (2°C to 8°C). Do not freeze. Can be kept at room temperature (below 40°C) for up to 5 days.
Administration	Oral or via feeding tube, once daily after a meal.
Developer	Genentech (a member of the Roche Group)
Approval Date	FDA approval in August 2020.

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