Duchenne Muscular Dystrophy

ACE Inhibitors May Help Slow Myocardial Fibrosis In Muscular Dystrophy

ACE Inhibitors May Help Slow Myocardial Fibrosis In Muscular Dystrophy

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Patients who received ACE inhibitors showed a slower progression of myocardial fibrosis and fewer cardiovascular events.

Eteplirsen Earns Accelerated Approval for Duchenne Muscular Dystrophy

Eteplirsen Earns Accelerated Approval for Duchenne Muscular Dystrophy

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Eteplirsen is the first drug approved in the US for the treatment of DMD.

Deflazacort Improves Muscle Strength, Functional Outcomes in Duchenne Muscular Dystrophy

Deflazacort Improves Muscle Strength, Functional Outcomes in Duchenne Muscular Dystrophy

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Over 52 weeks, patients taking deflazacort experienced improved muscle strength and less weight gain compared with prednisone.

Deflazacort Wins Priority Review for Duchenne Muscular Dystrophy Drug

Deflazacort Wins Priority Review for Duchenne Muscular Dystrophy Drug

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Deflazacort is intended to help improve muscle strength and other functional outcomes in DMD regardless of genetic etiology.

Duchenne Muscular Dystrophy Update: FDA Weighs Its Options

Duchenne Muscular Dystrophy Update: FDA Weighs Its Options

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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

FDA Delays Decision on Duchenne Muscular Dystrophy Drug

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This is the second extension requested by the FDA.

FDA Panel Votes Down Duchenne Muscular Dystrophy Drug Eteplirsen

FDA Panel Votes Down Duchenne Muscular Dystrophy Drug Eteplirsen

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The panel took a similar stance in the fall when it did not recommend Biomarin's drisapersen for the treatment of DMD.

FDA Denies NDA for Duchenne Muscular Dystrophy Drug Drisapersen

FDA Denies NDA for Duchenne Muscular Dystrophy Drug Drisapersen

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After a lukewarm review, the FDA has officially denied the NDA for drisapersen.

NDA Submitted for Duchenne Muscular Dystrophy Drug

NDA Submitted for Duchenne Muscular Dystrophy Drug

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Translarna (ataluren) is for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).

Screening for Duchenne Muscular Dystrophy May Produce False Negatives

Screening for Duchenne Muscular Dystrophy May Produce False Negatives

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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

Drisapersen Gets Lukewarm Review from FDA Committee

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The committee cited a lack of consistent clinical trial results as well as concerns over serious adverse events.

Genetic Target for Treating Duchenne Muscular Dystrophy Identified

Genetic Target for Treating Duchenne Muscular Dystrophy Identified

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Researchers have linked the Jagged1 gene to a much milder form of muscular dystrophy in golden retrievers.

Eteplirsen Improves Walking Ability in Duchenne Muscular Dystrophy

Eteplirsen Improves Walking Ability in Duchenne Muscular Dystrophy

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The FDA is set to make a decision on the NDA by late February, 2016.

Corticosteroid Regimen Delays Loss of Ambulation in Duchenne Muscular Dystrophy

Corticosteroid Regimen Delays Loss of Ambulation in Duchenne Muscular Dystrophy

A deflazacort regimen appeared to slow loss of ambulation, but not without several side effects.

Duchenne, Becker Muscular Dystrophy Affects 1 in 5,000 Boys in U.S.

Duchenne, Becker Muscular Dystrophy Affects 1 in 5,000 Boys in U.S.

The conditions are similar, but differ in rate of progression and age of onset.

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