Risdiplam
Mechanism :
This drug is a mRNA splicing modifier for the SMN2 protein. It prevents the destruction and increases the functional amount of this protein for patients with spinal muscular atrophy. Survival of motor neuron 2 (SMN2) mRNA modifier made to treat mutations in chromosome 5q. Which cause SMN protein deficiency in patients having spinal muscular atrophy.
Indication :
Dosing :
Spinal muscular atrophy:
More than 2 months of age:
Age more than 2 months to less than 2 years: 0.2 mg/kg Per Oral qDay.
Age more than years and weight less than 20 kg: 0.25 mg/kg Per Oral qDay.
Age more than years and weight less than 20 kg: 5 mg Per Oral qDay.
Adverse Effect :
Fever, diarrhea, rash, arthralgia, UTI, URTI, constipation, vomiting, penumonia, aphthous ulcers