Ever since the Jerry Lewis telethons began in the 1960s, millions of people have become familiar with an otherwise rare disease called muscular dystrophy (MD).
Aug 25, 2023
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Penn Medicine researchers have developed a new targeted approach to prevent a toxicity seen in the sensory neurons of dorsal root ganglia after gene therapy to treat neurological disorders. It's an important hurdle to clear, ...
Nov 11, 2020
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Each year, about 20,000 children are diagnosed with Duchenne muscular dystrophy, a rare genetic condition that causes progressive muscle weakness and other systemic damage.
Feb 18, 2022
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(Medical Xpress)—A team of researchers working in Japan has found that injecting a genetically engineered virus into the muscle of mice afflicted with two types of neuromuscular disease caused a reversal of symptoms. In ...
A new study published in the journal Nature Communications describes a breakthrough in research related to facioscapulohumeral muscular dystrophy (FSHD). The debilitating genetic disease - which has no approved treatment ...
Sep 15, 2017
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STRASBURG, PA- A new report has identified an alternative method to deliver nusinersen to patients with spinal muscular atrophy (SMA) using a subcutaneous intrathecal catheter system (SIC) configured by connecting an intrathecal ...
Aug 24, 2018
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Like a marching band falling out of step, muscle cells fail to perform in unison in patients with Duchenne muscular dystrophy. A new study in The Journal of Cell Biology reveals how this breakdown leads to the proliferation ...
Oct 14, 2014
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Spinal muscular atrophy (SMA) is a genetic disease that can leave infants with weak muscles and trouble breathing. Many with the disease die before age two. To help these patients, doctors need therapies that target the genetic ...
May 8, 2018
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Continuing a series of groundbreaking discoveries begun in 2010 about the genetic causes of the third most common form of inherited muscular dystrophy, an international team of researchers led by a scientist at Fred Hutchinson ...
Jan 12, 2012
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Obese mice that were fed a high-fat diet and that received prednisone one time per week had improved exercise endurance, got stronger, increased their lean body mass and lost weight, reports a new Northwestern Medicine study. ...
Apr 1, 2022
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