Duchenne


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Du·chenne

(dū-shen'),
References in periodicals archive ?
FDA's Office of Tissues and Advanced Therapies told Capricor Therapeutics (CAPR) that under the Regenerative Medicine Advanced Therapy (RMAT) designation, the agency will "work closely" with the company "to provide guidance on the subsequent development of CAP-1002" for the treatment of Duchenne muscular dystrophy, "including providing advice on generating the evidence needed to support approval of the drug in an efficient manner.
Researchers at the Eli and Edythe Broad Center at UCLA used the natural human development process as a guide, the researchers developed ways to mature muscle cells in the laboratory to create muscle fibers that restore dystrophin, the protein that is missing in the muscles of boys with Duchenne.
s (NASDAQ: CAPR) HOPE-1 clinical trial of its lead investigational product, CAP-1002, in boys and young men in advanced stages of Duchenne muscular dystrophy, the company said.
The Rare Pediatric Disease Designation adds to our previous Orphan Drug Designation for CAP-1002 for the treatment of Duchenne muscular dystrophy, which together underscore the urgent need for treatment options for this devastating rare disease as well as provide Capricor with certain incentives for their development," said Linda Marbn, Ph.
Exonics' CRISPR/Cas9 technology is a potential one-time treatment that would make a permanent correction of the mutation that causes Duchenne.
The report provides comprehensive information on the therapeutics under development for Duchenne Muscular Dystrophy, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
lt;BRyan, who suffers from Duchenne muscular dystrophy
The Jett Foundation, Michael's Cause and Ryan's Quest, nonprofits created by families of boys and young men with Duchenne, believe that their combined $15,000 gift will get promising treatments to patients quicker.
Last month it threw a surprise birthday party for William Magee, six, who has Duchenne Muscular Dystrophy - one of the biggest genetic killers among children.
For example, it's a lottery at the moment as to whether people with Duchenne can have access to a personal assistant.
We can't wait for our annual trip to Great Ormond Street as it's basically a 'one-stop shop' for managing Duchenne.
This phenomenon has been named as the Duchenne smile.

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