Category Archives: News from Other Sources

News gathered from external sources by our RSS aggregator.

A Protein Developed To Protect And Restore Nerve Cell Communications

A structure called “the microtubule network” is a crucial part of our nervous system. It acts as a transportation system within nerve cells, carrying essential proteins and enabling cell-to-cell communications. But in neurodegenerative diseases like Al…

Posted in News from Other Sources | Comments closed

Molecular Motors May Become Jammed, Playing A Role In The Development Of ALS

Slowdowns in the transport and delivery of nutrients, proteins and signaling molecules within nerve cells may contribute to the development of the neurodegenerative disorder ALS, according to researchers at the University of Illinois at Chicago College…

Posted in News from Other Sources | Comments closed

Many Children With Duchenne Muscular Dystrophy Could Benefit From Genetic Editing

Using a novel genetic ‘editing’ technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients…

Posted in News from Other Sources | Comments closed

Gene Therapy Helps Muscular Dystrophy Patients Breathe Easier

Children with a rare form of muscular dystrophy called Pompe disease often spend their days tethered to mechanical ventilators in order to breathe. But results from a new clinical trial at University of Florida Health show that gene therapy improved re…

Posted in News from Other Sources | Comments closed

Drug Benefits In Spinal Muscular Atrophy Animal Models

In a new publication that appears in Human Molecular Genetics, the laboratory of Christine DiDonato, PhD reports on their pharmacological characterization of the drug RG3039, demonstrating that it can extend survival and improve function in two spinal …

Posted in News from Other Sources | Comments closed

Engineered Stem Cell Advance Points Toward Treatment For ALS

Transplantation of human stem cells in an experiment conducted at the University of Wisconsin-Madison improved survival and muscle function in rats used to model ALS, a nerve disease that destroys nerve control of muscles, causing death by respiratory …

Posted in News from Other Sources | Comments closed

Exome Sequencing Technique Reveals Genetic Suspects In Sporadic Lou Gehrig’s Disease

Researchers at the Stanford University School of Medicine have identified mutations in several new genes that might be associated with the development of spontaneously occurring cases of the neurodegenerative disease known as amyotrophic lateral sclero…

Posted in News from Other Sources | Comments closed

Adult Mesenchymal Stem Cells Offer Hope For Treatment Of Amyotrophic Lateral Sclerosis

Transplantation of human stem cells in an experiment conducted at the University of Wisconsin-Madison improved survival and muscle function in rats used to model ALS, a nerve disease that destroys nerve control of muscles, causing death by respiratory …

Posted in News from Other Sources | Comments closed

Scientists Identify Another Gene Important To Morning Wake-Up Call

Northwestern University scientists have shown a gene involved in neurodegenerative disease also plays a critical role in the proper function of the circadian clock. In a study of the common fruit fly, the researchers found the gene, called Ataxin-2, ke…

Posted in News from Other Sources | Comments closed

Researchers Transform Adult Cells Into Early-Stage Nerve Cells, Bypassing The Pluripotent Stem Cell Stage

A University of Wisconsin-Madison research group has converted skin cells from people and monkeys into a cell that can form a wide variety of nervous-system cells – without passing through the do-it-all stage called the induced pluripotent stem cell, o…

Posted in News from Other Sources | Comments closed

  • Donate
  • Congenital Muscular Dystrophy

    A group of diseases causing muscle weakness at birth. Several defined genetic mutations cause muscles to break down faster than they can repair or grow. A child with CMD may have various neurological or physical impairments. Some children never gain the ability to walk, while others lose the ability as they grow older. Learn more...

  • Register Now!
  • Upcoming Fundraisers

    No events
  • Register Now!