Risdiplam Powder for Oral Solution 利司扑兰口服溶液

‌Lisapram oral solution‌ is a drug used to treat ‌spinal muscular atrophy (SMA). It improves motor function by regulating SMN2 gene splicing and increasing the production of functional SMN protein. The dosage should be adjusted according to the patient’s weight. It should be taken strictly according to the doctor’s instructions, and attention should be paid to possible side effects and contraindications.

1. Indications and mechanism of action
‌Indications‌: Mainly used for the treatment of ‌spinal muscular atrophy (SMA), including infantile, pediatric and adult patients. SMA is a rare genetic disease caused by SMN1 gene defects, which can lead to muscle weakness and motor function degeneration.
‌Mechanism of action‌: By acting on the ‌SMN2 gene, modifying its splicing process, increasing the production of full-length SMN protein, compensating for the missing function of the SMN1 gene, and delaying disease progression.
2. Usage and dosage
‌Dosage adjustment‌: The dosage should be strictly calculated according to the patient’s weight. It is usually taken orally once a day. The specific dosage is determined by the doctor after evaluation.
‌Dosage‌: The drug can be taken directly orally or mixed with a small amount of food. Make sure to swallow it completely to avoid dose loss.
‌Storage requirements‌: It needs to be stored in a refrigerator at 2-8℃ away from light. It must be used within the specified time after opening.
3. Precautions and side effects
‌Common side effects‌: May include fever, rash, diarrhea, etc.; in severe cases, elevated liver enzymes or allergic reactions may occur, and medical treatment should be sought in time.
‌Contraindications‌: It is contraindicated for those who are allergic to the drug ingredients; those with severe liver and kidney dysfunction need to be carefully evaluated.
‌Drug interactions‌: It may affect metabolism when used in combination with other drugs, and the doctor needs to be informed of other drugs being used.
‌Monitoring requirements‌: Liver function and SMN protein levels need to be checked regularly during treatment to evaluate efficacy and safety.
4. Applicable population and contraindications
‌Applicable population‌: Infants, children and adult patients diagnosed with SMA.
Those with SMN1 gene mutation confirmed by genetic testing and low SMN2 gene copy number.
‌Contraindications‌: Patients with severe liver function abnormalities. Those with specific metabolic disorders or drug allergy history.
V. Efficacy and long-term management
‌Efficacy evaluation‌: Early treatment can significantly improve exercise capacity and delay respiratory function decline, but individual differences are large.
‌Long-term management‌: It is necessary to combine multidisciplinary collaboration such as rehabilitation training and nutritional support, and regularly follow up and adjust the treatment plan.
‌Tips‌: Lisapran must be used under the guidance of a professional doctor, and the dose cannot be adjusted or stopped on your own. If abnormal symptoms occur during treatment, the medical team should be contacted in time.