Work

What Can We Learn From Clinical Trials of Exon Skipping for DMD?

Year: 2014

Type: article

Abstract: On 20 September 2013, GlaxoSmithKline (GSK) and Prosensa announced that GSK's Phase III clinical trial (NCT01254019) of Drisapersen, an exon skipping drug for Duchenne muscular dystrophy (DMD), failed to meet the primary endpoint of a statistically significant improvement in the 6 Minute Walking Distance Test (6MWT) compared to placebo.1 On 12 November 2013, Sarepta Therapeutics announced that the US Food and Drug Administration (FDA) has considered its application for accelerated approval of Eteplirsen as a DMD drug to be premature.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1038/mtna.2014.6

Authors: Qi-long Lu, Sebahattin Cirak, Terence Partridge

Institutions: Carolinas Medical Center, Children's National

Language: English

Topic: Muscle Physiology and Disorders

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900