Evrysdi vs. Zolgensma for Musculoskeletal Conditions: Important Differences and Potential Risks.

Evrysdi

(risdiplam)

Zolgensma

Summary for Evrysdi

Prescription only

Evrysdi (risdiplam) is used to treat a genetic condition called spinal muscular atrophy (SMA) in adults and children. It's a type of medication called an RNA splicing modifier. It works to help improve the motor function of people with SMA. Evrysdi (risdiplam) comes as a tablet and oral solution, and you take the medication by mouth once per day. But your specific dose depends on your age and weight. Side effects can include fever, diarrhea, and rash.

Indications for Evrysdi

Spinal muscular atrophy (SMA) in adults and children

Pros and Cons for Evrysdi

Pros

Taken by mouth once a day
Okay for people of all ages with SMA to take
Available as a tablet and a liquid

Cons

Can cause diarrhea
Must follow a lot of instructions to take the liquid safely (e.g., refrigeration, no mixing with food)
Can take months to notice better muscle strength

Common Side Effects for Evrysdi

Fever (22%)
Diarrhea (17%)
Rash (17%)

Warnings for Evrysdi

Possible interactions with other medications
Possible harm to an unborn baby
Possible fertility problems in males

Summary for Zolgensma

Prescription only

Zolgensma (onasemnogene abeparvovec or onasemnogene abeparvovec-xioi) is a medication that's used to treat a certain type of spinal muscular atropy in children under 2 years old. Zolgensma is given as a one-time infusion into the vein, but your child has to be monitored for at least 3 months after the treatment due to the risk of liver and clotting problems.

Indications for Zolgensma

Certain type of spinal muscular atropy (SMA) in children under 2 years old

Pros and Cons for Zolgensma

Pros

Convenient, one-time infusion treatment
Treats SMA by focusing on the genetic problem
Help babies achieve certain milestones, such as sitting by themselves for 30 seconds or more

Cons

Given as an infusion into the vein, so it might be uncomfortable for small children
Not approved to treat children with advanced SMA, such as complete paralysis of arms and legs, or using a permanent ventilator
Child must get regular lab work for at least 3 months after treatment