New hope for the treatment of Duchenne muscular dystrophy-Deflazacort

Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disease, with an incidence rate of 1/5000 to 1/3500 newborn male babies. The disease is caused by mutations in the DMD gene encoding dystrophin. Dystrophin deficiency affects the integrity and stability of the muscle membrane, leading to muscle fiber necrosis and regeneration, and muscle tissue fibrosis and fatification. The clinical manifestations are progressive muscle weakness, and most people die around the age of 20 due to respiratory and heart failure. The U.S. FDA approved (deflazacort) on February 9, 2017, for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years and older.

Deflazacort is a glucocorticoid that slows inflammation and reduces immune system activity. Glucocorticoids are often used to treat DMD around the world, but in the United States, this is the first time the FDA has approved glucocorticoids for the treatment of DMD, and it is also the first time the FDA has approved deflazacort for any therapeutic purpose.

Deflazacort is well absorbed orally and is rapidly converted into the active metabolite 21-OH deflazacort by plasma esterase in the body. The plasma concentration reaches a peak after 1.5 to 2 hours. The protein binding rate of deflazacort is 40%, mainly plasma proteins and platelets, and has no affinity for steroid transport globulin. Deflazacort can cross the blood-brain barrier at very low concentrations.

Adults and children 2 years of age and older take deflazacort 0.9 mg/kg orally daily. Common side effects of this drug are increased appetite, cough, weight gain, frequent urination, and upper respiratory tract infection. Finally, the editor of Medical Companion Travel would like to solemnly tell everyone that if the patient is currently taking the drug deflazacort, he must remember the doctor's instructions and take the medication according to the doctor's instructions. Only by carefully following the doctor's instructions can the drug itself be used to its greatest extent.

Note: The above information comes from the Internet and is compiled and edited by Medical Companion Travel (please correct me if there are any errors or omissions). It is only to provide information on the latest drugs on the market in the world and help Chinese patients understand the latest international new drug trends. It is only for internal discussion among medical staff and does not serve as any basis for medication. For specific medication guidelines, please consult the attending physician.