Research studies in mice using two markedly different strategies both resulted in increased life span
Below are highlights of two recent studies in spinal muscular atrophy (SMA), a disease in which the nerve cells that control muscles (motor neurons) in the spinal cord die, causing progressive weakness in the voluntary muscles.
An interview with MDA research grantee Matthew Disney explores his group's findings about small molecules as a potential treatment for myotonic dystrophy
In type 1 myotonic dystrophy (MMD1, or DM1), expansions of DNA on chromosome 19 known as CTG repeats are converted to expansions in RNA called CUG repeats, which are toxic to nerve and muscles cells in a variety of ways.
Several MDA-supported research teams are targeting the toxic CUG repeats, with the goal of either blocking their interaction with other cellular substances or destroying them...
An 11½-minute interview with researcher Stephen Tapscott explores recent findings about the molecular basis of facioscapulohumeral MD
A protein called DUX4, inappropriately produced ("expressed") in skeletal muscle fibers, is emerging as a major factor in facioscapulohumeral muscular dystrophy (FSHD).
In FSHD-affected muscles, full-length DUX4 protein disrupts numerous biochemical pathways that normally would help muscle cells survive, mature and develop specialized roles.
Molecular geneticist Stephen Wilton discusses his group's development of exon-skipping compounds to restore dystrophin production in Duchenne MD
A January 2012 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores the potential of a strategy called exon skipping as a treatment for Duchenne muscular dystrophy (DMD).
The podcast, which runs approximately 12 minutes, is part of a Nationwide Children's series called "This Month in Muscular Dystrophy."
Biologist Denis Guttridge discusses his team's work developing an NF-kappa B blocker as a potential treatment for Duchenne MD
A December 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores how an inhibitor of a protein called NF-kappa B has been beneficial in a mouse model of Duchenne muscular dystrophy (DMD) and is now being developed as a potential DMD treatment.
The podcast is part of a Nationwide Children's series called "This Month in Muscular Dystrophy."