Indication
Duchenne muscular dystrophy
Duchenne muscular dystrophy is a severe, rapidly progressive X-linked recessive neuromuscular disorder caused by mutations in the *DMD* gene that result in an absence or critical deficiency of the functional structural protein dystrophin. Read more about Duchenne muscular dystrophy
20 drugs 16 manufacturers
Drugs that treat it
Common Duchenne muscular dystrophy therapies.
EMFLAZA (deflazacort)
EMFLAZA (deflazacort)
EMFLAZA (deflazacort)
ELEVIDYS (delandistrogene moxeparvovec-rokl)
AGAMREE (vamorolone)
DUVYZAT (givinostat hydrochloride)
DEFLAZACORT
DEFLAZACORT
News & literature
Coming soonRelated codes
Coming soonRelated conditions & guidelines
Coming soonDuchenne muscular dystrophy is a severe, rapidly progressive X-linked recessive neuromuscular disorder caused by mutations in the *DMD* gene that result in an absence or critical deficiency of the functional structural protein dystrophin.
Primarily affecting males with early childhood onset, the lack of dystrophin compromises the sarcolemmal integrity of skeletal and cardiac muscle, rendering fibers susceptible to contraction-induced mechanical damage, chronic necrosis, inflammation, and progressive fibrofatty replacement.
Clinically, it presents with motor delays, proximal muscle weakness, and pseudohypertrophy, advancing inexorably toward loss of independent ambulation, restrictive pulmonary insufficiency, and dilated cardiomyopathy.
- medlineplus.gov
Duchenne muscular dystrophy is a severe, rapidly progressive X-linked recessive neuromuscular disorder caused by mutations in the *DMD* gene that result in an absence or critical deficiency of the functional structural protein dystrophin Primarily affecting males with early childhood onset, the lack of dystrophin compromises the sarcolemmal integrity of skeletal and cardiac muscle, rendering fibers susceptible to contraction-induced mechanical damage, chronic necrosis, inflammation, and progress...
- nih.gov
Duchenne muscular dystrophy is a severe, rapidly progressive X-linked recessive neuromuscular disorder caused by mutations in the *DMD* gene that result in an absence or critical deficiency of the functional structural protein dystrophin Primarily affecting males with early childhood onset, the lack of dystrophin compromises the sarcolemmal integrity of skeletal and cardiac muscle, rendering fibers susceptible to contraction-induced mechanical damage, chronic necrosis, inflammation, and progress...
- nih.gov
Duchenne muscular dystrophy is a severe, rapidly progressive X-linked recessive neuromuscular disorder caused by mutations in the *DMD* gene that result in an absence or critical deficiency of the functional structural protein dystrophin Primarily affecting males with early childhood onset, the lack of dystrophin compromises the sarcolemmal integrity of skeletal and cardiac muscle, rendering fibers susceptible to contraction-induced mechanical damage, chronic necrosis, inflammation, and progress...
Personalize your experience
Follow Duchenne muscular dystrophy and get updates as they land.
Already a member? Sign in
Follow an indication and get alerts when its drugs, labels, or guidelines change
Save any drug on this page to your favorites, synced across devices
AI-curated peer-reviewed research, summarized for your specialty
A morning digest of indication-relevant updates, when you want it
Sign in to follow this indication
Following an indication requires a free Physicians Office Resource account.