Emflaza⁚ A Closer Look at This DMD Treatment
Emflaza (deflazacort) is a corticosteroid medication specifically approved for the treatment of Duchenne Muscular Dystrophy (DMD) in patients aged 2 years and older. While it isn’t a cure for DMD, Emflaza can play a significant role in managing the disease by⁚
- Improving muscle strength⁚ Emflaza helps to increase muscle strength, which can improve mobility and function for individuals with DMD.
- Slowing disease progression⁚ The medication can help to slow the progression of muscle degeneration, potentially delaying the loss of ambulation and extending the period of independent living.
How Does Emflaza Work?
Emflaza is a pro-drug, meaning it is converted by the body into its active form, 21-desDFZ. This active metabolite works by⁚
* Decreasing inflammation⁚ Emflaza helps to reduce inflammation in muscles, which is a key factor contributing to muscle damage and weakness in DMD.
* Suppressing the immune system⁚ By suppressing the immune system, Emflaza may help to prevent further muscle damage caused by the body’s own immune response.
Administration and Dosage
Emflaza is available in two forms⁚
* Tablets⁚ Available in four different strengths, tablets can be taken whole or crushed and mixed with applesauce.
* Oral suspension⁚ This liquid form can be mixed with juice or milk and administered using the provided oral dispenser.
The recommended dosage is approximately 0.9 mg/kg/day٫ taken once daily. The specific dosage will be determined by your doctor based on your individual needs and response to the medication.
Important Considerations and Side Effects
Before starting Emflaza, it is crucial to discuss your medical history with your doctor, including any other medications you are taking, as drug interactions are possible. It is also important to be aware of potential side effects, which may include⁚
* Facial puffiness or Cushingoid appearance
* Weight gain and increased appetite
* Upper respiratory tract infections, cough, and colds
* Frequent daytime urination and unwanted hair growth
* Central obesity (increased fat around the abdomen)
* Changes in mood and behavior
* Increased risk of osteoporosis and bone fractures with long-term use
It is crucial to communicate any side effects you experience to your doctor, as they can help manage them or adjust your dosage if necessary.
Emflaza and DMD Management
Emflaza is a valuable tool in the management of DMD, offering the potential to improve muscle strength, slow disease progression, and enhance quality of life for individuals with this condition. However, it is essential to work closely with your doctor to determine if Emflaza is right for you and to monitor your progress and manage any potential side effects.
Remember, this information is not a substitute for professional medical advice. Always consult with your doctor regarding your specific situation and treatment options.