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110 results
  • Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. [Randomized Controlled Trial]
    Medicine (Baltimore) 2019; 98(26):e15858Alfano LN, Charleston JS, … Lowes LP
  • This analysis aims to describe the outcomes of two nonambulatory patients with Duchenne muscular dystrophy (DMD) who participated in two clinical studies. The two consecutive trials of eteplirsen (studies 201 and 202) were conducted in patients with DMD (N = 12) and confirmed genetic mutations amenable to exon 51 skipping.In study 201, 12 patients were randomized to receive once-weekly, double-bl…
  • What Is in the Myopathy Literature? [Review]
    J Clin Neuromuscul Dis 2019; 20(3):129-134Lacomis D, Abdel-Hamid HZ
  • We review the development of exon 51 skipping therapy with eteplirsen for Duchenne muscular dystrophy, including the recent report of long-term, sustained dystrophin production. Studies of the late-life health profile of patients with Duchenne muscular dystrophy, early detection of left ventricular systolic dysfunction, and caregiver burden are also covered. A study of skeletal muscle magnetic re…
  • Therapeutic Antisense Oligonucleotides Are Coming of Age. [Journal Article]
    Annu Rev Med 2019; 70:307-321Bennett CF
  • The first published description of therapeutic applications of antisense oligonucleotide (ASO) technology occurred in the late 1970s and was followed by the founding of commercial companies focused on developing antisense therapeutics in the late 1980s. Since the late 1980s, there has been steady progress in improving the technology platform, taking advantage of advances in oligonucleotide chemis…
  • Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches. [Review]
    J Pers Med 2019; 9(1)Shimizu-Motohashi Y, Komaki H, … Aoki Y
  • Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive muscle weakness, is caused by the absence or a decreased amount of the muscle cytoskeletal protein dystrophin. Currently, several therapeutic approaches to cure DMD are being investigated, which can be categorized into two groups: therapies that aim to restore dystrophin expression, and those that aim to compe…
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