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.

New zebrafish technique finds potential treatments for multiple diseases

Skeletal muscle has proved to be very difficult to grow in patients with muscular dystrophy and other disorders that degrade and weaken muscle. Researchers at Boston Children's Hospital's Stem Cell Program now report boosting muscle mass and reversing disease in a mouse model of Duchenne muscular dystrophy, using a "cocktail" of three compounds identified through a new rapid culture system. Adding the same compounds to stem cells derived from patients' skin cells, they then successfully grew human muscle cells in a dish.

8 Nov 2013

Scientists find key gene that activates muscle growth

For decades, scientists have searched for treatments for myopathies - genetic muscular diseases such as muscular dystrophy and ALS, also called Lou Gehrig's disease.

4 Nov 2013

New voting joystick could enable people with dexterity impairments to cast ballots independently

A voting joystick created at Michigan State University could eventually enable people with dexterity impairments, senior citizens and others to exercise their right to cast ballots independently.

30 Oct 2013

Researchers overcome obstacle to using viruses to deliver therapeutic genes

Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.

30 Oct 2013

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

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17 Oct 2013

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

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16 Oct 2013

New type of antibiotic kills multidrug-resistant germ common to health care settings

A new type of antibiotic called a PPMO, which works by blocking genes essential for bacterial reproduction, successfully killed a multidrug-resistant germ common to health care settings, UT Southwestern Medical Center researchers report.

16 Oct 2013

NIH selects Salk scientist for EUREKA grant

The National Institutes of Health has selected Axel Nimmerjahn for a highly competitive EUREKA (Exceptional Unconventional Research Enabling Knowledge Acceleration) grant. Dr. Nimmerjahn is an Assistant Professor in the Waitt Advanced Biophotonics Center and holds the Richard Allan Barry Developmental Chair.

13 Oct 2013

CommerceWest Bank CEO receives Robert Ross Founders Award

Ivo Tjan, Chairman and CEO of CommerceWest Bank, was honored as the Robert Ross Founders Award Recipient at the Augie's Quest 8th Annual Tradition of Hope Gala.

13 Oct 2013

Research points to causes, potential treatment approaches for systemic sclerosis

Using mice, lab-grown cells and clues from a related disorder, Johns Hopkins researchers have greatly increased understanding of the causes of systemic sclerosis, showing that a critical culprit is a defect in the way certain cells communicate with their structural scaffolding

10 Oct 2013

Three Penn Medicine researchers awarded grants for neuromuscular disease research

The Muscular Dystrophy Association has awarded research grants totaling over $1,000,000 to three Penn Medicine researchers: James Shorter, PhD, associate professor of Biochemistry and Biophysics; Hansell Stedman, MD, associate professor of Surgery; and Lee Sweeney, PhD, director of the Center for Orphan Disease Research and Therapy.

9 Oct 2013

NeuroVigil opens satellite research lab at NASA to develop assistive technologies for ALS

NeuroVigil, Inc. is setting to collaborate with a number of partners in the Silicon Valley where it is opening a satellite research laboratory at NASA Research Park in Mountain View, CA, to develop assistive technologies for individuals suffering from ALS and other conditions affecting the central nervous system.

9 Oct 2013

Quintiles and MDA partner to develop U.S. Neuromuscular Disease Registry

Quintiles and the Muscular Dystrophy Association (MDA) today announced a new partnership to develop and implement the U.S. Neuromuscular Disease Registry, a patient registry that will play an important role in determining effective treatments for people with muscular dystrophy and related muscle diseases.

9 Oct 2013

Professor receives $1.3M EUREKA grant to explore communication between cytoplasm and nucleus

University of Massachusetts Medical School Professor and Vice Chair of Neurobiology Vivian Budnik, PhD, has received a four-year, $1.3 million EUREKA (Exceptional Unconventional Research Enabling Knowledge Acceleration) grant from the National Institute of Neurological Disorders and Stroke to explore a novel mechanism of communication between the cytoplasm and the nucleus, called nuclear envelope budding, that may lead to new understandings for various tissue dystrophies and aging disorders.

8 Oct 2013

ISIS conducts phase 2 study of ISIS-SMNRx in infants with type I spinal muscular atrophy

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it dosed the first infant in the 12 mg dose group in the Phase 2 study evaluating ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA).

4 Oct 2013

Scientists unravel secrets of mechanism that snips our genes

Certain diseases such as cystic fibrosis and muscular dystrophy are linked to genetic mutations that damage the important biological process of rearranging gene sequences in pre-messenger RNA, a procedure called RNA splicing.

27 Sep 2013

Stem cell lines are ideal research tools for designing models to understand disease progression

Scientists from King's College London have announced that 16 human embryonic stem (hES) cell lines have been approved by the US National Institutes of Health (NIH) and placed on their Stem Cell Registry, making them freely available for federally-funded research in the USA.

25 Sep 2013

Isis Pharmaceuticals announces preliminary data from ISIS-SMNRx Phase 1 study in infants with SMA

Isis Pharmaceuticals, Inc. announced today that follow-up preliminary data from a single dose, open-label Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy, show that most SMA children receiving the two highest doses of the drug (6 mg and 9 mg) continued to show improvements in muscle function tests up to 14 months after a single injection of the drug.

19 Sep 2013

VBP15 drug shows early promise for treatment of Duchenne muscular dystrophy

A preclinical study led by researchers at Children's National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy.

18 Sep 2013

Perkins+Will completes UF's Clinical and Translational Research Building

The Miami office of global architecture and design firm, Perkins+Will, recently completed a major healthcare, education and research project, the University of Florida's Clinical and Translational Research Building.

11 Sep 2013

Muscular Dystrophy News and Research

Further Reading

New zebrafish technique finds potential treatments for multiple diseases

Scientists find key gene that activates muscle growth

New voting joystick could enable people with dexterity impairments to cast ballots independently

Researchers overcome obstacle to using viruses to deliver therapeutic genes

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

Jain Foundation Steps Up Efforts To Find Cure For Muscular Dystrophy

New type of antibiotic kills multidrug-resistant germ common to health care settings

NIH selects Salk scientist for EUREKA grant

CommerceWest Bank CEO receives Robert Ross Founders Award

Research points to causes, potential treatment approaches for systemic sclerosis

Three Penn Medicine researchers awarded grants for neuromuscular disease research

NeuroVigil opens satellite research lab at NASA to develop assistive technologies for ALS

Quintiles and MDA partner to develop U.S. Neuromuscular Disease Registry

Professor receives $1.3M EUREKA grant to explore communication between cytoplasm and nucleus

ISIS conducts phase 2 study of ISIS-SMNRx in infants with type I spinal muscular atrophy

Scientists unravel secrets of mechanism that snips our genes

Stem cell lines are ideal research tools for designing models to understand disease progression

Isis Pharmaceuticals announces preliminary data from ISIS-SMNRx Phase 1 study in infants with SMA

VBP15 drug shows early promise for treatment of Duchenne muscular dystrophy

Perkins+Will completes UF's Clinical and Translational Research Building

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