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.

Breakthrough study promises to bring gene-editing technology much closer to therapeutic reality

Imagine a future where a guided biomachine put into your body seeks out defective gene sequences in each cell and edits in the correct information with precision accuracy.

13 Apr 2018

Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy

Cells made by fusing a normal human muscle cell with a muscle cell from a person with Duchenne muscular dystrophy --a rare but fatal form of muscular dystrophy -- were able to significantly improve muscle function when implanted into the muscles of a mouse model of the disease.

16 Mar 2018

UCLA-developed nanospears could make gene therapies safer, faster and more cost-effective

UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called "nanospears" for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.

15 Mar 2018

New guidelines intend to provide template for optimal, most up-to-date DMD care

Diagnostic and treatment advances are helping patients with Duchenne muscular dystrophy - one of nine major types of muscular dystrophy that affects males - live into their 30s and beyond, raising challenges in such areas as education, vocation, levels of independence, personal relationships, emotional health, and intimacy.

13 Mar 2018

NIH-funded researchers use CRISPR to rapidly find new genetic suspects behind ALS, FTD

NIH-funded researchers at Stanford University used the gene editing tool CRISPR-Cas9 to rapidly identify genes in the human genome that might modify the severity of amyotrophic lateral sclerosis and frontotemporal dementia caused by mutations in a gene called C9orf72.

13 Mar 2018

Study reveals key function of ALS-linked protein

The protein FUS, whose mutation or disruption causes many cases of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), works as a central component of one of the most important regulatory systems in cells, according to a new study in Molecular Cell from scientists at Johns Hopkins Bloomberg School of Public Health.

8 Mar 2018

Salk researchers uncover power switch for muscles

If you've ever wondered how strenuous exercise translates into better endurance, researchers at the Salk Institute may have your answer. In a study published in the journal Cell Reports on March 6, 2018, scientists in Ronald Evans' lab have shown that the protein ERRγ (ERR gamma) helps deliver many of the benefits associated with endurance exercise.

6 Mar 2018

Plymouth researcher receives grant to further investigate causes of hereditary neuro-tumors

The University of Plymouth will be continuing its research into the advancement of neuro-tumor treatments thanks to more than £100,000 from Great Ormond Street Hospital Children's Charity and Sparks, the medical research charity.

5 Mar 2018

Leading consultants from the North East putting rare diseases expertise on world stage

This Rare Disease Day (28 February), the Academic Health Science Network, is shining a spotlight on the region’s healthcare pioneers chosen to lead three major European rare disease networks.

1 Mar 2018

Cell therapy for heart disease caused by muscular dystrophy also improves limb strength, study shows

Injections of cardiac progenitor cells help reverse the fatal heart disease caused by Duchenne muscular dystrophy and also lead to improved limb strength and movement ability, a new study shows.

23 Feb 2018

New tool measures cell strength 100 times faster than current technologies

Engineers, doctors and scientists at UCLA and Rutgers University have developed a tool that measures the physical strength of individual cells 100 times faster than current technologies.

12 Feb 2018

Cholesterol and other lipids in the blood play role in many diseases

Lipids are fatty molecules that play important signaling and storage roles in the body, but having an excess of some lipids, like cholesterol, is a risk factor for many metabolic diseases.

8 Feb 2018

Researchers test effectiveness of investigational drug in treating diastolic dysfunction of the heart

Researchers at the University of Colorado School of Medicine have identified a potential treatment target for patients with a common type of heart failure.

8 Feb 2018

USC scientists uncover cellular mechanism responsible for ALS and frontotemporal dementia

Scientists have for the first time discovered a mechanism that limits the number of "cellular janitors" in the nervous system, leading to increased risk for two neurodegenerative diseases: amyotrophic lateral sclerosis and frontotemporal dementia, according to a Keck School of Medicine of USC study published today in Nature Medicine.

7 Feb 2018

New CRISPR gene-editing technique can correct majority of mutations that cause DMD

Scientists have developed a CRISPR gene-editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center.

6 Feb 2018

Researchers find new method to overcome major challenge of stem cell therapy

Researchers have discovered a new technique that overcomes one of the major challenges of stem cell therapy.

25 Jan 2018

Scientists create artificial human muscles

Researchers have successfully created artificial human muscles in the laboratories that are tiny but fully functional. These small muscles actually respond to the nerve stimulations, says the latest study reporting this scientific breakthrough.

10 Jan 2018

FDA chief says he’s open to rethinking incentives on orphan drugs

The commissioner of the Food and Drug Administration questions whether the right financial incentives are in place for drugmakers who develop orphan drugs for rare diseases.

22 Dec 2017

UCLA researchers reveal reversibility of Friedreich's ataxia in mouse models

In children and adults with Friedreich's ataxia, an inherited disease that causes damage to the nervous system, a loss of coordination typically progresses to muscle weakness and can involve vision, diabetes, and other problems over the course of several years.

20 Dec 2017

Study reveals biology behind why muscle stem cells respond differently to aging or injury

A new study led by researchers at Sanford Burnham Prebys Medical Discovery Institute describes the biology behind why muscle stem cells respond differently to aging or injury.

15 Dec 2017