Myotonic Dystrophy News and Research

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Biogen Idec And Isis Ink Fourth Deal In Two Years

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9 Sep 2013

Novel way to dramatically raise potency of drug candidates targeting RNA

Scientists from the Jupiter campus of The Scripps Research Institute (TSRI) have shown a novel way to dramatically raise the potency of drug candidates targeting RNA, resulting in a 2,500-fold improvement in potency and significantly increasing their potential as therapeutic agents.

6 Aug 2013

TSRI scientists identify small molecules to control genetic defect responsible for muscular dystrophy

For the first time, scientists from the Florida campus of The Scripps Research Institute have identified small molecules that allow for complete control over a genetic defect responsible for the most common adult onset form of muscular dystrophy.

28 Jun 2013

New hope for patients with myotonic dystrophy

There's hope for patients with myotonic dystrophy. A new small molecule developed by researchers at the University of Illinois has been shown to break up the protein-RNA clusters that cause the disease in living human cells, an important first step toward developing a pharmaceutical treatment for the as-yet untreatable disease.

2 May 2013

Clinical trial signals new era in treatment of neurodegererative disorders

The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis - blocking production of a mutant protein that causes an inherited form of the progressive neurodegererative disease - may be a first step towards a new era in the treatment of such disorders.

4 Apr 2013

Systemic delivery of antisense oligonucleotides neutralizes mutant RNA toxicity in mice with DM1

By targeting the specific mutation that causes the hereditary neuromuscular disease myotonic dystrophy, it is possible to neutralize the mutant RNA toxicity and minimize or even eliminate the disabling symptoms of the disease.

25 Feb 2013

Scientists design small molecules that recognize myotonic dystrophy-associated RNAs

Scientists from the Florida campus of The Scripps Research Institute (TSRI) have developed a new approach to alter the function of RNA in living cells by designing molecules that recognize and disable RNA targets. As a proof of principle, in the new study the team designed a molecule that disabled the RNA causing myotonic dystrophy.

21 Dec 2012

Isis, Biogen Idec partner to discover and develop antisense drugs against neuromuscular disorders

Biogen Idec and Isis Pharmaceuticals, Inc. today announced that they have entered into a global collaboration agreement under which the companies will discover and develop antisense drugs against three undisclosed targets to treat neurological or neuromuscular disorders.

11 Dec 2012

Researchers hold new implications for pathogenesis of myotonic dystrophy

An important breakthrough could help in the fight against myotonic dystrophy. The discovery, recently published in the prestigious scientific journal Cell, results from an international collaboration between researchers at the IRCM, the Massachusetts Institute of Technology (MIT), the University of Southern California and Illumina.

13 Sep 2012

Scripps Florida designs new compound that shows promise against tremor ataxia syndrome

Scientists on the Florida campus of The Scripps Research Institute have designed a compound that shows promise as a potential therapy for one of the diseases closely linked to fragile X syndrome, a genetic condition that causes mental retardation, infertility, and memory impairment, and is the only known single-gene cause of autism.

6 Sep 2012

Scientists reveal how TREX proteins act as passport for transfer of cell blueprints

Decoded process could hold the key to future treatments for a wide range of chronic health problems including Motor Neuron Disease, myotonic dystrophy and a wide range of cancers, University of Sheffield scientists have revealed.

16 Aug 2012

Molecular missteps disrupt brain function in adult-onset muscular dystrophy

The molecular missteps that disrupt brain function in the most common form of adult-onset muscular dystrophy have been revealed in a new study published by Cell Press. Myotonic dystrophy is marked by progressive muscle wasting and weakness, as well as excessive daytime sleepiness, memory problems, and mental retardation.

10 Aug 2012

Myotonia less important to patients with myotonic dystrophy than other symptoms

Complex, multi-system diseases like myotonic dystrophy - the most common adult form of muscular dystrophy - require physicians and patients to identify which symptoms impact quality of life and, consequently, what treatments should take priority. However, a new study out this month in the journal Neurology reveals that there is often a disconnect between the two groups over which symptoms are more important, a phenomenon that not only impacts care but also the direction of research into new therapies.

26 Jul 2012

Keck Foundation awards UF $1M to study how proteins in cells are made

The Center for NeuroGenetics at the University of Florida has received a competitive $1 million grant from the W.M. Keck Foundation to study a new idea that challenges conventional understanding of how proteins in cells are made, and their impact on inherited diseases.

16 Apr 2012

Invasive strategy benefits patients with myotonic dystrophy type 1 neuromuscular disorder

Patients with a cardiac irregularity and myotonic dystrophy type 1 (a severe neuromuscular disorder with a high risk of sudden death) who received an invasive treatment strategy that included testing of their heart's electrical conduction system and if needed, implantation of a device such as a pacemaker, had an associated higher rate of 9-year survival compared to patients treated noninvasively, according to a study in the March 28 issue of JAMA.

28 Mar 2012

Scientists design compounds that act against myotonic dystrophy type 1 RNA

While RNA is an appealing drug target, small molecules that can actually affect its function have rarely been found. But now scientists from the Florida campus of The Scripps Research Institute have for the first time designed a series of small molecules that act against an RNA defect directly responsible for the most common form of adult-onset muscular dystrophy.

23 Feb 2012

Myotonic Dystrophy News and Research

Biogen Idec And Isis Ink Fourth Deal In Two Years

Novel way to dramatically raise potency of drug candidates targeting RNA

TSRI scientists identify small molecules to control genetic defect responsible for muscular dystrophy

New hope for patients with myotonic dystrophy

Clinical trial signals new era in treatment of neurodegererative disorders

Systemic delivery of antisense oligonucleotides neutralizes mutant RNA toxicity in mice with DM1

Scientists design small molecules that recognize myotonic dystrophy-associated RNAs

Isis, Biogen Idec partner to discover and develop antisense drugs against neuromuscular disorders

Researchers hold new implications for pathogenesis of myotonic dystrophy

Scripps Florida designs new compound that shows promise against tremor ataxia syndrome

Scientists reveal how TREX proteins act as passport for transfer of cell blueprints

Molecular missteps disrupt brain function in adult-onset muscular dystrophy

Myotonia less important to patients with myotonic dystrophy than other symptoms

Keck Foundation awards UF $1M to study how proteins in cells are made

Invasive strategy benefits patients with myotonic dystrophy type 1 neuromuscular disorder

Scientists design compounds that act against myotonic dystrophy type 1 RNA

Prosensa secures €23M in new equity financing

People with myotonic muscular dystrophy more likely to develop cancer

Insight into neurodegenerative diseases

Medication used to treat heart arrhythmias also reduces myotonic dystrophy: Researchers

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