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CAS NO.1825352-65-5
Quality Manufacturer Customized Supply Reliable Quality 1825352-65-5 Risdiplam, accept customization.
Risdiplam acts as a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier, increasing the levels of functional SMN protein. Part 2 of ongoing trials will provide important data on the long-term efficacy and safety of risdiplam in infants with Type 1 SMA.
Risdiplam, developed by Roche, PTC Therapeutics Inc, and the SMA Foundation, received its first approval in the USA in August 2020 for the treatment of SMA in patients 2 months of age and older. Risdiplam is the only oral option among the three therapies approved for SMA. It is suitable for use in adults, children, and infants.
For patients between 2 months and 2 years: 0.2 mg/kg once daily after meals.
For patients older than 2 years:
Weighing less than 20 kg: 0.25 mg once daily after meals.
Weighing 20 kg or more: 5 mg once a day.
Onset of Action:
Time to reach maximum plasma concentration after oral administration: 1 to 4 hours.
In clinical trials, SMN protein levels in the blood more than doubled within four weeks of treatment.
It's crucial for patients to follow the prescribed dosage and administration instructions and report any side effects to their healthcare provider. Regular monitoring is essential to assess treatment efficacy.