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.

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

Researchers discover how high glucose levels in pregnancy affect baby's heart

Researchers at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have discovered how high glucose levels -- whether caused by diabetes or other factors -- keep heart cells from maturing normally.

12 Dec 2017

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

SLU scientists provide promising approach in designing new drugs for DMD

In a paper published in the Nature journal Scientific Reports, Saint Louis University researchers report that a new drug reduces fibrosis (scarring) and prevents loss of muscle function in an animal model of Duchenne muscular dystrophy (DMD), providing a promising approach in designing new medications for those suffering from DMD.

8 Dec 2017

UT Southwestern researchers develop improved gene-editing technique to correct DMD mutations

Regenerative medicine researchers at UT Southwestern Medical Center developed an improved and simplified gene-editing technique using CRISPR/Cas9 tools to correct a common mutation that causes Duchenne muscular dystrophy.

30 Nov 2017

New initiative aims to improve chances of medicines receiving positive HTA decisions

A unique initiative launched today by muscular dystrophy charity Duchenne UK is set to radically simplify the way necessary evidence is generated for submissions made to health technology appraisals bodies such as the National Institute for Health and Care Excellence and the Haute Autorité de Santé.

22 Nov 2017

New research shows how a gene can fuel obesity in mice

Obesity is often attributed to a simple equation: People are eating too much and exercising too little. But evidence is growing that at least some of the weight gain that plagues modern humans is predetermined.

16 Nov 2017

Clinical trial results could lead to treatment of heart disease in Duchenne muscular dystrophy patients

After boys and young men with Duchenne muscular dystrophy received cardiac progenitor cell infusions, medical tests indicated that the patients' hearts appeared improved, results from a new study show. Patients in the study also scored higher on arm strength tests after receiving the cell infusions.

15 Nov 2017