Duchenne muscular dystrophy occurs in boys and is characterized by progressive muscle degeneration and weakness leading to a decline in respiratory function. Strategies to arrest this severe progressive deterioration are ...
Jul 9, 2019
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Gene therapy—for so long something that belonged to the future—has just hit the streets.
Jun 5, 2019
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(HealthDay)—The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy (SMA), the U.S. Food and Drug Administration announced Friday.
May 28, 2019
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There is now further evidence that a drug that is effective in treating the rare muscle-wasting disease spinal muscular atrophy (SMA) early in life may be associated with improvement in older children, according to a study ...
Apr 24, 2019
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The Stentrode is a device that is placed inside a blood vessel of the brain located in an area that controls movement (motor cortex). It is the only investigational technology of its kind that does not require open brain ...
Apr 8, 2019
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Duchenne muscular dystrophy (DMD) is a severe muscle disease that causes progressive muscle weakening and degeneration.
Apr 5, 2019
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Researchers in Florida never expected this much success with a drug they're developing to treat muscular dystrophy.
Mar 27, 2019
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'Organoids' that mimic the developing spinal cord could assist research and drug development for neurodegenerative diseases such as spinal muscular atrophy and amyotrophic lateral sclerosis.
Mar 18, 2019
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By sequencing the entire genomes of tumor cells from six people with a rare cancer of the nose and sinus cavity, Johns Hopkins researchers report they unexpectedly found the same genetic change¾one in a gene involved in ...
Feb 27, 2019
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Facioscapulohumeral muscular dystrophy (FSHD) is the most prevalent dominantly inherited muscular dystrophy in the world. To date, there are no pharmacologic treatments available for the more than 850,000 people affected ...
Nov 16, 2018
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