Miscellaneous musculoskeletal disorders:
Indications for: EVRYSDI
Spinal muscular atrophy (SMA).
Adults and Children:
Must be administered immediately after drawn up into the oral syringe. Take once daily after a meal at same time each day. May be given via nasogastric or gastrostomy tube if unable to swallow. <2months: 0.15mg/kg. 2months–<2yrs: 0.2mg/kg. ≥2yrs (<20kg): 0.25mg/kg; (≥20kg): 5mg. Breastfed infants: administer after breastfeeding; do not mix with formula or milk.
Avoid inhalation and direct contact with skin or mucous membranes with dry powder and constituted solution. Hepatic impairment: avoid. Pregnancy: exclude status prior to initiation. Advise females of reproductive potential to use effective contraception during and for ≥1 month after the last dose. Possible male infertility. Nursing mothers.
Survival motor neuron 2 (SMN2) splicing modifier.
Avoid concomitant with MATE substrates (eg, metformin); if unavoidable, monitor for drug-related toxicities; consider dose reduction of substrate.
Late-onset: fever, diarrhea, rash; Infantile-onset: also upper respiratory tract infection, pneumonia, constipation, vomiting.
Fecal (53%), renal (28%).
Apparent clearance: 2.45 L/h (for a 31.3 kg patient).
Half–life: 50 hours.
Generic Drug Availability:
Bottle (80mL)—1 (w. adapter, oral syringes)