Advances in the Treatment of Duchenne Muscular Dystrophy: New and Emerging Pharmacotherapies.
Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disease that primarily affects young males. Patients with DMD are unable to produce dystrophin, a crucial protein found in myocytes, leading to a loss of muscle support and integrity. Corticosteroids are the standard supportive treatment for DMD; however, there is a high demand to expand the number of safe, effective pharmacologic options. Recently a surge of new therapeutics for DMD is offering hope to patients. A variety of these new medications, such as stop codon readthrough agents, exon-skipping agents, and utrophin modulators, aim to replace dystrophin in myocytes. Other new therapeutics aim to prevent or repair muscle damage caused by the absence of dystrophin. This review provides an update on the medications being investigated in DMD.
School of Pharmacy, Regis University Rueckert-Hartman College for Health Professions, Denver, Colorado.
Andrea M
AM
Reinig
School of Pharmacy, Regis University Rueckert-Hartman College for Health Professions, Denver, Colorado.
Sara
S
Mirzaei
School of Pharmacy, Regis University Rueckert-Hartman College for Health Professions, Denver, Colorado.
Daniel J
DJ
Berlau
D015195:Drug Design; D015507:Drugs, Investigational / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D016189:Dystrophin / Q000378:metabolism; D005938:Glucocorticoids; D006801:Humans; D008297:Male; D032342:Muscle Cells / Q000187:drug effects / Q000378:metabolism; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology
D015507:Drugs, Investigational; D016189:Dystrophin; D005938:Glucocorticoids
ataluren; drisapersen; dystrophin; eteplirsen; exon-skipping; ezutromid; utrophin