Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.

UofL researchers discover mechanism involved in skeletal muscle repair

Researchers at the University of Louisville have discovered a mechanism involved in skeletal muscle repair that may enable clinicians to boost the effectiveness of adult stem cell therapies for diseases such as muscular dystrophy.

1 Dec 2015

Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner

In addition to their well established cholesterol lowering benefits, statins also have potent anti-inflammatory, anti-fibrotic and antioxidant effects, which continue to be identified in a wide range of diseases.

30 Nov 2015

Researchers use magic-angle-spinning NMR to unveil structure of CAP-Gly protein

A latticework of tiny tubes called microtubules gives your cells their shape and also acts like a railroad track that essential proteins travel on. But if there is a glitch in the connection between train and track, diseases can occur.

26 Nov 2015

Duchenne muscular dystrophy could directly affect muscle stem cells

A new study from The Ottawa Hospital and the University of Ottawa is poised to completely change our understanding of Duchenne muscular dystrophy and pave the way for far more effective treatments.

18 Nov 2015

Researchers identify factors that increase risk of cardiac arrest during pediatric spine surgeries

Although the vast majority of pediatric spine surgeries are safe, a handful of neuromuscular conditions seem to fuel the risk of cardiac arrest during such operations, according to research led by investigators at the Johns Hopkins Children's Center.

18 Nov 2015

Drug compounds target multiple pathways associated with myotonic dystrophy type 1

Efforts to treat myotonic dystrophy type 1, the most common form of muscular dystrophy, are in their infancy. In a new study, researchers report they have added new capabilities to an experimental drug agent that previously defeated only one of DM1's many modes of action. Their retooled compounds interrupt the disease's pathology in three ways.

10 Nov 2015

Simple, low risk blood test can detect foetal blood group and genetic conditions in unborn babies

Research into a simple, accurate and low risk blood test that can detect foetal blood group, sex, and genetic conditions in unborn babies has been published in the international scientific journal, Clinical Chemistry.

9 Nov 2015

U-M study suggests new therapeutic strategy for treating Duchene muscular dystrophy

Every heart beat and step in our daily lives is dependent on the integrity of muscles and the proteins that keep them strong and free of injury as they contract and relax.

27 Oct 2015

MU researchers successfully treat dogs with DMD, plan for human clinical trials

Muscular dystrophy, which affects approximately 250,000 people in the U.S., occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way to successfully treat the most common form of the disease, Duchenne Muscular Dystrophy (DMD), which primarily affects boys. Now, a team of University of Missouri researchers have successfully treated dogs with DMD and say that human clinical trials are being planned in the next few years.

23 Oct 2015

Genetic mutation linked to ALS, FTD prevents proteins from entering and exiting the cell's nucleus

Three teams of scientists supported by the National Institutes of Health showed that a genetic mutation linked to some forms of amyotrophic lateral sclerosis (ALS) and frontotemporal degeneration (FTD) may destroy neurons by disrupting the movement of materials in and out of the cell's nucleus, or command center where most of its DNA is stored.

19 Oct 2015

UTHealth biochemists expose structures of IP3R calcium channels

Calcium is a crucial element in the body that controls thought, movement and other bodily functions. These events are directed by specialized proteins called ion channels that allow the flow of calcium ions in and out of cells and among cell compartments. For years, scientists have been unsure how calcium ion channels function.

15 Oct 2015

New hope for patients with Duchenne muscular dystrophy

New hope for Duchenne muscular dystrophy (DMD) patients. A mouse genetic study in PLoS Medicine reports that targeting the P2RX7 gene, a purinoreceptor, may halt the progression of DMD.

14 Oct 2015

Researchers identify new therapeutic target for Duchenne muscular dystrophy

Scientists have found what could one day lead to a promising treatment for those with a crippling disease, Duchenne muscular dystrophy.

14 Oct 2015

New breakthrough may provide key to understanding how to treat Muscular Dystrophy

New research published today in the journal eLife has demonstrated a new method for observing the behaviour of the protein Dystrophin in a living animal cell, in real-time. This breakthrough may provide a key to understanding how to treat the genetic disease, Muscular Dystrophy.

14 Oct 2015

Quest Diagnostics scientists demonstrate new molecular combing technique that predicts cystic fibrosis, muscular dystrophy

Genetic testing for mutations beyond those currently recommended in medical guidelines may aid the identification of more couples at risk of having a child with cystic fibrosis, according to a study presented today at the 2015 American Society of Human Genetics Annual Meeting, which runs through October 10 in Baltimore, Maryland.

From Quest Diagnostics 13 Oct 2015

Study: RNA editing technique shows promise in treating rare, severe form of muscular dystrophy

An RNA editing technique called "exon skipping" has shown preliminary success in treating a rare and severe form of muscular dystrophy that currently has no treatment, based on a new study from Northwestern Medicine and the University of Chicago. Children with the disease lose significant muscle strength early in life.

13 Oct 2015

Invitae announces dramatic expansion of genetic testing menu

Invitae Corporation (NYSE: NVTA), a genetic information company, has announced that it has more than doubled the size of its genetic testing platform to include more than 600 genes and will begin releasing the new content between now and the end of the year. Invitae is immediately expanding its menu with dozens of new test panels for hereditary cancer, cardiovascular, neuromuscular, pediatric and other rare disorders.

7 Oct 2015

Tarix Orphan's TXA127 granted FDA Fast Track Designation for treatment of DMD patients

Tarix Orphan LLC, a privately held biopharmaceutical company focused on the treatment of rare neuromuscular disorders and connective tissue diseases, today announced that the U.S. Food and Drug Administration has granted Fast Track Designation to TXA127 (angiotensin 1-7) to reduce skeletal muscle damage and fibrosis and thereby improve muscle strength in Duchenne Muscular Dystrophy (DMD) patients.

6 Oct 2015

Research shows why microgravity leads to muscle loss

People can easily feel the presence - or absence - of gravity. Our individual cells actually may be able to sense gravity, too, and that ability could play a role in the loss of muscle that occurs when humans spend time in space.

1 Oct 2015

AMO Pharma raises $25 million in private equity financing

AMO Pharma Limited, a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, announced today that the company has raised $25 million in private equity financing from Woodford Investment Management.

30 Sep 2015

Muscular Dystrophy News and Research

Further Reading

UofL researchers discover mechanism involved in skeletal muscle repair

Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner

Researchers use magic-angle-spinning NMR to unveil structure of CAP-Gly protein

Duchenne muscular dystrophy could directly affect muscle stem cells

Researchers identify factors that increase risk of cardiac arrest during pediatric spine surgeries

Drug compounds target multiple pathways associated with myotonic dystrophy type 1

Simple, low risk blood test can detect foetal blood group and genetic conditions in unborn babies

U-M study suggests new therapeutic strategy for treating Duchene muscular dystrophy

MU researchers successfully treat dogs with DMD, plan for human clinical trials

Genetic mutation linked to ALS, FTD prevents proteins from entering and exiting the cell's nucleus

UTHealth biochemists expose structures of IP3R calcium channels

New hope for patients with Duchenne muscular dystrophy

Researchers identify new therapeutic target for Duchenne muscular dystrophy

New breakthrough may provide key to understanding how to treat Muscular Dystrophy

Quest Diagnostics scientists demonstrate new molecular combing technique that predicts cystic fibrosis, muscular dystrophy

Study: RNA editing technique shows promise in treating rare, severe form of muscular dystrophy

Invitae announces dramatic expansion of genetic testing menu

Tarix Orphan's TXA127 granted FDA Fast Track Designation for treatment of DMD patients

Research shows why microgravity leads to muscle loss

AMO Pharma raises $25 million in private equity financing

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