Myotonic Dystrophy News and Research

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Scientists reveal ability to reverse Huntington's disease-causing DNA repeat mutations in the lab

Neurodegenerative diseases, like Huntington's disease and myotonic dystrophy, are often referred to as DNA repeat diseases, named because of long repeated sequences in the DNA of patients. Increasing repeat expansion length in the affected tissues contribute to earlier age of disease onset and worsen the progression and severity of the disease over time.

27 Feb 2020

Potential drug targets cause of adult-onset muscular dystrophy

People diagnosed with myotonic dystrophy type 1 have difficulty unclenching muscles due to a genetic defect that generates toxic material within their cells.

1 Apr 2019

Creative solutions to enhance patient's point of view in neuromuscular research

The old-fashioned paternalistic relationship between doctors and patients has gradually evolved into a more collaborative one in the era of patient-centered medicine.

7 Feb 2019

New method to speed up genetic diagnosis of Huntington’s disease

Elongated segments of DNA cause Huntington's disease and certain other disorders of the brain. Researchers funded by the SNSF have developed a method to determine the length of the mutated genes quickly and easily.

10 Jan 2019

New mouse model shows potential for rapid identification of promising muscular dystrophy therapies

A Massachusetts General Hospital research team has created a new mouse model of a common form of muscular dystrophy with the potential of rapidly distinguishing promising therapeutic drugs from those unlikely to be successful.

13 Dec 2018

Asuragen launches assay to help diagnose Steinert’s Disease

Asuragen, Inc., a molecular diagnostics company delivering easy-to-use products for complex testing in genetics and oncology, today announced the CE marking and launch of the AmplideX DM1 Dx Kit, which simplifies the analysis of repeat expansions within the DMPK gene and is intended to aid in the diagnosis of Myotonic Dystrophy Type I (DM1), also known as Steinert's Disease.

4 Dec 2018

First bifacial molecule can invade double-stranded DNA or RNA

Carnegie Mellon University researchers have developed a synthetic molecule that can recognize and bind to double-stranded DNA or RNA under normal physiological conditions. The molecule could provide a new platform for developing methods for the diagnosis and treatment of genetic conditions.

13 Nov 2018

Study highlights role of non-mutational mechanism in myotonic dystrophy, other types of arrhythmias

A team of researchers at Baylor College of Medicine reveals new insights into what can be causing life-threatening cardiac-related events in myotonic dystrophy type 1.

28 Sep 2018

Urine exRNA may be source of biomarkers for muscular dystrophy

Massachusetts General Hospital researchers have found that extracellular RNA in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.

26 Sep 2018

Study finds that several existing anti-cancer drugs may work in part by binding to RNA

Bolstering the notion that RNA should be considered an important drug-discovery target, scientists at Scripps Research have found that several existing, FDA-approved anti-cancer drugs may work, in part, by binding tightly to RNA, the regulators of the basic activities of life within cells.

30 Jun 2018

Researchers discover new approach for treatment of myotonic dystrophy

Researchers at the Translational Genomics laboratory of Valencia University and the INCLIVA health research institute have just discovered a new approach for the treatment of myotonic dystrophy, a rare and incurable neuromuscular disease which does not have specific treatment.

27 Jun 2018

Researchers reveal pathological mechanisms in congenital myotonic dystrophy

Myotonic dystrophy (DM) is the most common form of genetic muscular dystrophy that begins in adulthood. DM is characterized by progressive muscle wasting and weakness. People with this disorder often have prolonged muscle contractions (myotonic) and are not able to relax certain muscles after use.

12 Dec 2017

Research brings scientists one step closer to understanding development of ALS

University of Michigan-led research brings scientists one step closer to understanding the development of neurodegenerative disorders such as ALS.

8 Dec 2017

Protein changes essential for normal adult muscle function

Researchers at Baylor College of Medicine have shed light on the process that guides the maturation of newborn muscles into adult, fully functional organs.

14 Aug 2017

New version of gene editing technique corrects molecular mistakes in RNA-based diseases

Until recently, the CRISPR-Cas9 gene editing technique could only be used to manipulate DNA. In a 2016 study, University of California San Diego School of Medicine researchers repurposed the technique to track RNA in live cells in a method called RNA-targeting Cas9 (RCas9).

11 Aug 2017

Scientists uncover new gene therapy treatment routes for motor neurone disease

Scientists investigating the genetic causes and altered functioning of nerve cells in motor neurone disease (MND) have discovered a new mechanism that could lead to fresh treatment approaches for one of the most common forms of the disease.

14 Jul 2017

Researchers discover basic genetic mechanism that causes congenital myotonic dystrophy

For years, the underlying process that causes a debilitating muscle disorder in infants and young children has been largely unknown.

11 Jul 2017

TSRI researchers develop new drug candidates to target prostate and triple negative breast cancers

Scientists on the Florida campus of The Scripps Research Institute (TSRI) have designed two new drug candidates to target prostate and triple negative breast cancers.

9 Mar 2017

TSRI scientists develop new methods to design precision medicines against disease-causing RNAs

Scientists on the Florida campus of The Scripps Research Institute have developed broad methods to design precision medicines against currently incurable diseases caused by RNA.

20 Dec 2016

Tufts scientists discover reason for incidence of genetic error that causes many disorders

Tufts University researchers have discovered a possible explanation for the occurrence of a genetic error that causes over a dozen neuromuscular and neurodegenerative disorders, including Huntington's disease, myotonic dystrophy and forms of spinocerebellar ataxia.

6 Dec 2016