Muscular Dystrophy

Facioscapulohumeral Muscular Dystrophy Treatment Gets Fast Track Status

Facioscapulohumeral Muscular Dystrophy Treatment Gets Fast Track Status

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ACE-083 is currently being studied in a Phase 2 trial for FSHD and another for Charcot-Marie-Tooth (CMT) disease.

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.

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.

NIH Launches Genome Sequencing Program for Common, Rare Diseases

NIH Launches Genome Sequencing Program for Common, Rare Diseases

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The CCDG and CMG programs aim to discover the genes and genomic variants that cause or contribute to various diseases.

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.

Whole Exome Sequencing Aids Diagnosis of Limb-Girdle Muscular Dystrophy

Whole Exome Sequencing Aids Diagnosis of Limb-Girdle Muscular Dystrophy

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Overall, whole exome sequencing achieved a 45% success rate in difficult-to-diagnose cases of LGMD.

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.

Cosmetic Lip Augmentation May Relieve Facial Paralysis

Cosmetic Lip Augmentation May Relieve Facial Paralysis

The procedure may have benefits for those with stroke, Bell's palsy, and muscular dystrophy.

New Guidelines for Congenital Muscular Dystrophy

New Guidelines for Congenital Muscular Dystrophy

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The guidelines emphasize a multidisciplinary approach to the diagnosis and treatment of CMDs.

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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