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Muscular Dystrophy News
August 14, 2020

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Top Headlines
 

New Treatment for Common Form of Muscular Dystrophy Shows Promise in Cells, Animals

Researchers have designed a potential new treatment for one of the most common forms of muscular dystrophy. Medical researchers have created and ...

New Gene Correction Therapy for Duchenne Muscular Dystrophy

Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of 12 and reducing life expectancy. Researchers have ...

CRISPR Halts Duchenne Muscular Dystrophy Progression in Dogs

Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a new study that provides a strong indication ...

'CRISPR-Gold' Fixes Duchenne Muscular Dystrophy Mutation in Mice

Scientists have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne ...
Latest Headlines
updated 10:28pm EDT

Earlier Headlines
 

Genomic Cut and Paste Using a Class 1 CRISPR System

Repairing faulty genes to prevent and cure disease is something researchers have been working towards for many years. While Class 2 CRISPR systems show great promise as gene editing tools in human ...

Researchers Developing New 'DNA Stitch' to Treat Muscular Dystrophy

A new therapeutic being tested is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy (DMD). The treatment -- a cocktail of ...

Cheaper Drug Just as Effective Protecting Heart in Duchenne Muscular Dystrophy

A new clinical trial found a cost-effective generic drug works just as well as a more expensive drug in preserving heart function in boys with Duchenne muscular ...

New Research Identifies Gene That Hides Cancer Cells from Immunotherapy

A team has identified a gene that could make immunotherapy treatments, specifically checkpoint inhibitors, work for a wider variety of cancer patients. The study found that when the DUX4 gene is ...

Worm Study Sparks Hope for Slowing Muscle Decline

Muscle decline caused by ageing and certain diseases could be dramatically slowed by stopping a chain reaction that damages cells, new research ...

Lithium Boosts Muscle Strength in Mice With Rare Muscular Dystrophy

Researchers have found that lithium improves muscle size and strength in mice with a rare form of muscular dystrophy that causes weakness in the shoulders and hips. The findings could lead to a drug ...

Novel Protein Degradation Pathway

A research team how a type of protein that is embedded in the inner nuclear membrane clears out of the system once it has served its ...

Muscle Gene Mutations Implicated in Human Nasal/sinus Cancer

By sequencing the entire genomes of tumor cells from six people with a rare cancer of the nose and sinus cavity, researchers report they unexpectedly found the same genetic change -- one in a gene ...

New Drug for Duchenne Muscular Dystrophy Clears Phase 1 Clinical Trial Testing in Boys

Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic ...

New Insight Into Use of Cell Replacement Therapies to Treat Muscular Dystrophies

The University of Minnesota Medical School continues its legacy of advancing cell replacement therapies with a scientific breakthrough that highlights the promise of cell therapies for muscular ...

New Research Insights Hold Promise for Kids With DMD

Prednisone, the current standard of care used to treat kids with Duchenne muscular dystrophy (DMD), reduces chronic inflammation but has harsh side effects. Eplerenone, a heart failure drug, is used ...

Gene Therapy Cassettes Improved for Muscular Dystrophy

Experimental gene therapy cassettes for Duchenne muscular dystrophy have been modified to deliver better performance. The cassettes, which carry the therapy into muscle cells, contain newer versions ...

Discovery Points to Innovative New Way to Treat Duchenne Muscular Dystrophy

Researchers have discovered a new way to treat the loss of muscle function caused by Duchenne muscular dystrophy in animal models of the disease. The team restored muscle stem cell function that is ...

Stem Cell Researchers Develop Promising Technique to Generate New Muscle Cells in Lab

To help patients with muscle disorders, scientists have engineered a new stem cell line to study the conversion of stem cells into ...

A Molecule for Fighting Muscular Paralysis

Myotubular myopathy is a severe genetic disease that leads to muscle paralysis. Although no treatment currently exists, researchers have identified a molecule that not only greatly reduces the ...

Investigational Steroid Mirrors Prednisone's Benefits While Taming Its Side Effects

A head-to-head trial comparing the decades-old steroid, prednisone, and a promising new steroid, vamorolone, finds both act on the same key set of genetic pathways involved in controlling ...

Duchenne Muscular Dystrophy: How Muscle Cells Journey to the Dark Side

Answers to treating muscular dystrophies could lie in better understanding muscle repair -- which resembles a delicate cellular dance choreographed by special cells called fibro-adipogenic ...

Diseased Heart Muscle Cells Have Abnormally Shortened Telomeres

People with a form of heart disease called cardiomyopathy have abnormally short telomeres in heart muscle cells responsible for contraction, according to a new ...

Stabilizing Dysferlin-Deficient Muscle Cell Membrane Improves Muscle Function

In a head-to-head trial between the conventional glucocorticoid, prednisolone, and a modified glucocorticoid, vamorolone, in experimental models of LGMD2B, vamorolone improved dysferlin-deficient ...

Muscle Stem Cells Derived from Teratomas

Researchers have developed a process to regenerate skeletal muscle cells in mice with muscular ...

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