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.
How Scientists are Using iPSCs to Develop New Therapies

How Scientists are Using iPSCs to Develop New Therapies

Gene Therapy Shows Promise for Muscular Dystrophy Patients

Gene Therapy Shows Promise for Muscular Dystrophy Patients

Disclosing the Key Role of Mitofusin 2 Cellular Makeup in Interconnecting Organelles Within Cells

Disclosing the Key Role of Mitofusin 2 Cellular Makeup in Interconnecting Organelles Within Cells

Review: Role of Alternative Splicing Defects in Major Neurodegenerative Diseases

Review: Role of Alternative Splicing Defects in Major Neurodegenerative Diseases

Scarring on the Extracellular Matrix Causes Muscles to Stop Working in Duchenne Muscular Dystrophy

Scarring on the Extracellular Matrix Causes Muscles to Stop Working in Duchenne Muscular Dystrophy

Optimized Genome-Editing Method Vastly Reduces Mutations

Optimized Genome-Editing Method Vastly Reduces Mutations

The relationship between cancer and DMD gene expression discovered

The relationship between cancer and DMD gene expression discovered

Scientists receive $2.5 million NIH grant to define the role of lipid droplets in muscle stem cell function

Scientists receive $2.5 million NIH grant to define the role of lipid droplets in muscle stem cell function

Toxic protein likely to be the cause of two rare genetic disorders identified

Toxic protein likely to be the cause of two rare genetic disorders identified

New center at UCLA focuses on the microbiome’s role in disease prevention

New center at UCLA focuses on the microbiome’s role in disease prevention

Structure of amyloid fibers formed by the protein hnRNPDL-2 determined

Structure of amyloid fibers formed by the protein hnRNPDL-2 determined

New genetic condition that raises vulnerability to opportunistic infections

New genetic condition that raises vulnerability to opportunistic infections

RNA-targeting strategy to repair the genetic cause of ALS and dementia

RNA-targeting strategy to repair the genetic cause of ALS and dementia

Parents transmit genetic and epigenetic information to their offspring

Parents transmit genetic and epigenetic information to their offspring

National DNA Day 2022: PacBio HiFi Sequencing Contributes to First Complete Human Genome

National DNA Day 2022: PacBio HiFi Sequencing Contributes to First Complete Human Genome

Cell-derived therapy may help treat ventricular arrhythmia

Cell-derived therapy may help treat ventricular arrhythmia

Scientists use mRNA delivery to enhance muscle power

Scientists use mRNA delivery to enhance muscle power

Cellular therapy leads to meaningful functional improvements in DMD patients

Cellular therapy leads to meaningful functional improvements in DMD patients

Experts introduce new gene therapy approach for muscle weakness

Experts introduce new gene therapy approach for muscle weakness

Gene writing helps develop advanced therapies safely and effectively

Gene writing helps develop advanced therapies safely and effectively