Introduction
Duchenne muscular dystrophy (DMD) is a challenging condition. It weakens muscles over time. Emflaza helps slow this process. This article explains what Emflaza is, how it works, and what you should know.
What Is Emflaza?
Emflaza is a drug for DMD. It’s a corticosteroid named deflazacort. Doctors prescribe it for kids and adults with DMD, starting at age 2.
- Generic Name: Deflazacort
- Brand Name: Emflaza
- Forms: Tablets (6 mg to 86 mg) and liquid (22.75 mg/ml)
- FDA Approval: Approved in 2017 for ages 5+, updated to 2+ in 2019
It’s the first corticosteroid the FDA approved for DMD. That makes it special.
How Does Emflaza Work?
DMD occurs when your body misses a protein called dystrophin. Without it, muscles weaken and break down. Emflaza steps in to help.
Here’s what it does:
- It lowers inflammation, slowing muscle damage.
- It calms the immune system, which can overact in DMD.
Picture it as a guard. It doesn’t replace the protein, but it shields muscles from harm.
Benefits of Emflaza
Emflaza won’t cure DMD. But it offers real help. Check out its perks.
- Slows Muscle Loss: Kids stay strong longer.
- Boosts Movement: Makes walking and climbing easier.
- Delays Wheelchair Use: Some kids walk for extra years.
A study showed boys on Emflaza walked longer than those on placebo. That boosts life quality.
How to Take Emflaza
Using Emflaza is simple. Your doctor picks the dose. Here’s the rundown.
- Dose: Based on weight, often 0.9 mg per kg daily.
- Timing: Take it once daily, with or without food.
- Missed a Dose? Take it when you recall. Skip if the next dose is near.
Don’t quit cold turkey. That can cause issues. Your doctor will guide you to stop safely.
Side Effects and Risks
Emflaza has side effects. Most are mild. Some need attention.
Common Side Effects
- Weight gain
- Puffy face
- Bigger appetite
- Cough or cold
These often fade. Tell your doctor if they bug you.
Serious Side Effects
- Weak Bones: Risk of breaks rises.
- High Blood Sugar: Look for thirst or frequent peeing.
- Mood Swings: You might feel cranky or sad.
Call your doctor quick if you get bad stomach pain or blurry vision.
Who Should Use Emflaza?
Emflaza suits people with DMD, ages 2 and up. But it’s not for all. Skip it if:
- You’re allergic to deflazacort.
- You have a fungal infection.
- Your vaccines aren’t current—check with your doctor.
Tell your doctor your health details. They’ll confirm it’s okay for you.
What Studies Show
Research supports Emflaza. It works well. Here’s proof.
- A 12-week study with 196 boys showed stronger muscles with Emflaza.
- Another test found boys walked longer on it.
- Over time, it helps heart and lung health.
Still, it’s not flawless. Side effects can be rough. Your doctor will balance the good and bad.
Off-Label Uses
Emflaza is built for DMD. Some doctors try it elsewhere, but proof is thin.
- Other Muscle Diseases: Like Becker’s muscular dystrophy.
- Inflammation Issues: Think asthma or arthritis.
These aren’t FDA-backed. We need more studies.
FAQ: Your Questions Answered
Got curiosities? Here’s what people ask most.
- How fast does it kick in?
Some feel it in weeks. Big changes take months. - Safe with other drugs?
Ask your doctor. Some combos can clash. - Is it pricey?
Often, yes. Look into insurance or aid programs. - Can I quit if I feel good?
No way. Stopping can worsen DMD. Check with your doctor.
Conclusion
Emflaza fights DMD’s muscle damage. It’s not a fix, but it keeps strength longer. Side effects exist, yet many find it worthwhile. Got DMD? Talk to your doctor about Emflaza.