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Summit Corporation plc (‘Summit’ or ‘the Company’) SUMMIT OUTLINES CLINICAL DEVELOPMENT PLANS FOR UTROPHIN MODULATOR PROGRAMME FOR DUCHENNE MUSCULAR DYSTROPHY First patient clinical trials of SMT C1100 expected to start […]
Duchenne muscular dystrophy (DMD) patients suffer from cardiorespiratory complications in which progressive respiratory failure is the most common cause of mortality, and cardiomyopathy accounts for 20%-40% of all deaths. Inhibitors of the renin-angiotensin-aldosterone system (with or without beta blockers) are used clinically to delay progressive ventricular dysfunction.
The use of antisense oligonucleotides (AON) to induce exon skipping in precursor mRNA is at the forefront of clinical research for the treatment of DMD. The most advanced programs from Sarepta Therapeutics and GSK/Prosensa target exon 51, and are currently in Phase II and Phase III clinical trails in the US and the rest of the world. Their approval is anticipated within the near future.
Strategies that could enhance or improve the efficiency of the current (and future) exon skipping drugs to produce novel dystrophin have been sought by a number of research groups over the last few years. Now a recent report by Kendall et al. describes the identification of a compound that enhances the efficiency of exon skipping in vitro and in vivo. The compound, the approved drug Dantrolene, works in concert with the exon skipping drug to restore dystrophin levels in the mdxmice and also in reprogrammed myotubes from DMD patients. This research offers insight into the exon skipping process, and could provide a fast track opportunity to use an approved drug to enhance the efficiency of current exon skipping drugs in future clinical trials.
A recent placebo-controlled clinical study on the effects of the PDE5A inhibitor drug Tadalafil (Cialis), demonstrated beneficial effects on muscle blood flow in a model of muscle ischemia in men with Becker muscular dystrophy who harbor a mutation in which the neuronal nitric oxide synthase is absent. The result reinforce earlier findings in mice and Duchene patients and highlight the role of this pathway in muscle homeostasis.
Directors of the California stem cell agency today deferred action on a $20 million proposal that was rejected by its grant reviewers and sent it back for more consideration.
Sarepta Therapeutics (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced that treatment with its exon-skipping compound, eteplirsen, achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT), over a placebo/delayed treatment cohort in a Phase IIb trial in Duchenne muscular dystrophy (DMD) patients.
A recent paper from Elisabeth Rumeur and colleagues (http://www.ojrd.com/content/pdf/1750-1172-7-45.pdf) introduces a new database (http://edystrophin.genouest.org/) that is made freely available for public access. It contains information from 945 clinical reports.
The eDystrophin database compliments two other databases of DMD human mutations: the Leiden Muscular Dystrophy database and the UMD-DMD French database. The eDystrophin database is specifically dedicated to providing information about in-frame mutations (deletions, duplications and substitutions) of the DMD gene and the consequences of these alterations from a clinical perspective and a protein structure perspective.
The database is user friendly and informative and should be a source of much needed information for researchers, parents and patients alike.
Researchers at Stanford University have for the first time sequenced the genome of an unborn baby using only a blood sample from the mother.
Myostatin is both a regulator of muscle growth and a stimulator of muscle fibroblasts to proliferate; and there is an accumulating body of evidence that demonstrates inhibition of myostatin/ActRIIB signaling can ameliorate the pathology and function of dystrophic muscle in preclinical models of Duchene muscular dystrophy.