Muscular Dystrophy News and Research

RSS

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.

Researchers identify new components of genetic pathway that controls neural stem cells

Can one feel too attached? Does one need to let go to mature? Neural stem cells have this problem, too. As immature cells, neural stem cells must stick together in a protected environment called a niche in order to divide so they can make all of the cells that populate the nervous system. But when it's time to mature, or differentiate, the neural stem cells must stop dividing, detach from their neighbors and migrate to where they are needed to form the circuits necessary for humans to think, feel and interact with the world.

26 Apr 2012

Positive results from Repligen's RG3039 Phase 1 study for spinal muscular atrophy

Repligen Corporation today announced positive results from a Phase 1 study to evaluate the pharmacokinetic (PK) and safety profile of RG3039, a novel small molecule drug candidate for the potential treatment of spinal muscular atrophy (SMA).

25 Apr 2012

Myomatrix 2012 conference to take place from April 22-24

The nation's leading scientists and clinicians exploring treatment breakthroughs for congenital muscular dystrophy will convene April 22-24 on the campus of the University of Nevada, Reno, where research by Dean Burkin has led to a potential therapy.

19 Apr 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

KLF15 gene governs the body's ability to burn fat during exercise

While exercise is accepted universally as the most beneficial prescription physicians can write for patients, little is known about the molecular mechanisms that generate its widespread health benefits. Researchers from Case Western Reserve School of Medicine have shed light on this mystery by discovering that a genetic factor, Kruppel-like Factor 15 (KLF15), governs the body's ability to burn fat during exercise.

10 Apr 2012

Scientists discover why rapamycin causes diabetic-like symptoms in patients

Scientists at Dana-Farber Cancer Institute have discovered why diabetic-like symptoms develop in some patients given rapamycin, an immune-suppressant drug that also has shown anti-cancer activity and may even slow ageing.

4 Apr 2012

New method to easily identify cause of rare diseases

Sanford Health Children's Research Center researcher, Kyle Roux, PhD, has revealed a new method of research that will allow the cause of disease—particularly rare diseases—to become more easily identified.

23 Mar 2012

New method for in vivo study of unknown selenium metabolism

Spanish and Danish researchers have developed a method for the in vivo study of the unknown metabolism of selenium, an essential element for living beings. The technique can help clarify whether or not it possesses the anti-tumour properties that have been attributed to it and yet have not been verified through clinical trials.

22 Mar 2012

TACT believes that Halo's HT-100 may effectively treat DMD

Halo Therapeutics, LLC, a clinical-stage biopharmaceutical company developing novel therapeutics for rare fibrotic diseases, announced today that the TREAT-NMD Advisory Committee on Therapeutics (TACT) has reviewed HT-100, Halo's drug candidate for Duchenne muscular dystrophy (DMD), and believes the compound has potential based on the proposed mechanism.

21 Mar 2012

New three-step process for DMD newborn screening

Investigators at Nationwide Children's Hospital, working with the DNA Sequencing Core Facility at the University of Utah, have developed an approach to newborn screening (NBS) for the life-threatening genetic disorder, Duchenne muscular dystrophy (DMD) and potentially other muscular dystrophies.

20 Mar 2012

Researchers discover molecular mechanism responsible for low back pain

Italian researchers at the Catholic University of Sacred Heart in Rome found an important molecular mechanism responsible for low back pain and other acute vertebral problems like cervical axial pain, all due to aging and degeneration of the vertebral column.

14 Mar 2012

Human diseases with Drosophila counterparts discussed at annual conference

More than two-thirds of human genes have counterparts in the well-studied fruit fly, Drosophila melanogaster, so although it may seem that humans don't have much in common with flies, the correspondence of our genetic instructions is astonishing. In fact, there are hundreds of inherited diseases in humans that have Drosophila counterparts.

12 Mar 2012

Many children with life limiting conditions have been underestimated

The number of children with conditions such as muscular dystrophy, neurodegenerative disorders or severe cerebral palsy who are surviving into adulthood has been underestimated, a new study shows.

12 Mar 2012

New DNA-reading technology could lead to correct genetic diagnosis for muscle-wasting diseases

Scientists at The University of Nottingham have used a revolutionary new DNA-reading technology for a research project that could lead to correct genetic diagnosis for muscle-wasting diseases.

6 Mar 2012

IOM report identifies options that could further improve information obtained from pediatric studies

Federal laws that motivate or require drug and biologic developers to conduct pediatric studies have yielded beneficial information to guide the use of medications in children, says a new report by the Institute of Medicine. Still, studies involving children continue to be limited, especially in certain areas such as medications' use in newborns and long-term safety and effectiveness in children.

1 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

Study examines abdomyosarcoma growth in mouse models of muscular dystrophy

Inflammation, cell division and cell differentiation that occur during skeletal muscle regeneration may provide an ideal environment for the highly malignant tumor, rhabdomyosarcoma to arise. These are the findings from a Nationwide Children's Hospital study that examined rhabdomyosarcoma growth in mouse models of muscular dystrophy.

23 Feb 2012

Brief 10-minute massage helps reduce inflammation in muscle

Researchers at McMaster University have discovered a brief 10-minute massage helps reduce inflammation in muscle.

1 Feb 2012

Prosensa secures €23M in new equity financing

Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet needs, announced today that it has raised €23 million in new equity financing.

25 Jan 2012

Three-parent children may be born in near future in an effort to combat mitochondrial diseases

In the next couple of years the scientific community may witness creation of children with genetic material from three parents. British scientists want to start testing a new IVF technique to try to eradicate incurable genetic diseases such as muscular dystrophy. The concept took a leap forward yesterday after the Government announced a public consultation on whether the law should be changed to allow the technique.

22 Jan 2012