| خلاصه مقاله | A multidisciplinary medical, surgical, and rehabilitative approach targeting the symptoms of DMD can alter the natural course of the disease, improving longevity and quality of life
Other members of the DMD team may include:
Pediatric Orthopedist
Pediatric Gastroenterologist/Hepatologist
Geneticist
Primary Care Physician/Other Healthcare Professionals
Physiotherapist
Todate there is no curative treatment for DMD.
Currently, there are only supportive treatments that include:
Glucocorticoids and similar drugs
Gene therapy
Cell therapy
The standard dosage is 0.75 mg/kg/d for prednisone and0.9 mg/kg/d for deflazacort (maximum daily dosage in patients weighing more than 40 kg are 30 mg prednisoneand 36 mg deflazacort)
The considerable advantage of these pharmacological strategies is that they apply to all DMD patients irrespective of their mutation type, and Compared with gene-targeted and cell-based therapies, these molecular drugs with histories of clinical application show less unexplored safety problems.
In several clinical trials, both daily prednisone and daily deflazacort were more effective than intermittent prednisone.
Non-steroidal advances in treating Duchenne
givinostat (Duvyzat)
The U.S. FDA Approved drug in March 2024, Duvyzat (givinostat), an oral medication for the treatment of DMD in patients 6 years of age and older. It is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.
It is a histone deacetylase (HDAC) inhibitor that works by targeting pathogenic processes to reduce inflammation and muscle loss.
Duvyzat’s DMD treatment efficacy was evaluated in a randomized, double-blind, placebo-controlled 18-month phase 3 study. All participants continued to receive a standard-of-care steroid regimen throughout the study. |