Skeletal Maturation Delayed in Glucocorticoid-Treated Patients With Duchenne Muscular Dystrophy
Skeletal maturation is significantly delayed in boys with glucocorticoid-treated Duchenne muscular dystrophy.
Duchenne Muscular Dystrophy
Spironolactone Noninferior in Duchenne Muscular Dystrophy
In a head-to-head study of mineralocorticoid receptor antagonists, spironolactone was found to be noninferior to eplerenone for slowing the progression of heart damage in boys with Duchenne muscular dystrophy.
Orphan Drug Designation Granted to Pamrevlumab for Duchenne Muscular Dystrophy
The Food and Drug Administration (FDA) has granted Orphan Drug designation to pamrevlumab (FibroGen), for the treatment of patients with Duchenne muscular dystrophy (DMD).
FDA to Review DMD Treatment Golodirsen
Duchenne affects an estimated 1 in approximately every 3,500-5,000 males born worldwide.
Eteplirsen Earns Accelerated Approval for Duchenne Muscular Dystrophy
Eteplirsen is the first drug approved in the US for the treatment of DMD.
Duchenne Muscular Dystrophy Update: FDA Weighs Its Options
The DMD space is becoming increasingly crowded as the FDA works to identify a drug worth approving.
FDA Delays Decision on Duchenne Muscular Dystrophy Drug
This is the second extension requested by the FDA.
NDA Submitted for Duchenne Muscular Dystrophy Drug
Translarna (ataluren) is for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
Screening for Duchenne Muscular Dystrophy May Produce False Negatives
Researchers recommend that follow-up protocols be developed for those with elevated CK but negative DMD gene testing.
Drisapersen Gets Lukewarm Review from FDA Committee
The committee cited a lack of consistent clinical trial results as well as concerns over serious adverse events.
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