PTC Therapeutics Continue To Fight For Duchenne Muscular Dystrophy Community
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PTC Therapeutics Continue To Fight For Duchenne Muscular Dystrophy Community

Even after the third denial from the U.S. FDA, Food and Drug Administration, PTC Therapeutics Incorporation will not stop fighting for the drug Translarna that they believe will help patients…

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Turning Skin Cells Into Muscle Cells: Treatment for Muscular Disorders
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Turning Skin Cells Into Muscle Cells: Treatment for Muscular Disorders

There is potential for a new treatment for Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness.To learn more about this…

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Irish Boy with Duchenne Muscular Dystrophy Might Not Have Time to Wait for Country to Cover Treatment

For now, Ann Marie Harte’s five-year-old son, Lewis, can still walk. He can no longer run, and he has a hard time with the stairs. The trampoline exhausts him, and…

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Fighting for a Cure for Duchenne Muscular Dystrophy: British Family Raises Millions
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Fighting for a Cure for Duchenne Muscular Dystrophy: British Family Raises Millions

What can one little boy and his family do to fight a rapidly debilitating and (currently) incurable disease such as Duchenne muscular dystrophy? A lot. The diagnosis of a loved…

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PBS Documentary “The Gene Doctors” Brings Rare Treatments to the Public Eye
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PBS Documentary “The Gene Doctors” Brings Rare Treatments to the Public Eye

Earlier this month, PBS released the "The Gene Doctors." The hour-long documentary follows the recent emergence of gene therapy as a novel method of treating rare diseases. The film discusses…

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Safer, More Efficient CRISPR Tech Offers Possible Duchenne Muscular Dystrophy Treatment

We've written about CRISPR technology before, and it's potential for treating sickle cell anemia. But in a latest study done at the University of California, Berkeley, sponsored by the NIH,…

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Petition Aims to Heighten Awareness of World Duchenne Muscular Dystrophy Day
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Petition Aims to Heighten Awareness of World Duchenne Muscular Dystrophy Day

Duchenne muscular dystrophy (DMD) is a heartbreaker for the the 250,000 families worldwide whose sons are born with this debilitating, progressive disease. It is rare, and yet the ripples of its…

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A Drug Developed in Australia For Duchenne Muscular Dystrophy Hasn’t Been Approved There
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A Drug Developed in Australia For Duchenne Muscular Dystrophy Hasn’t Been Approved There

Duchenne muscular dystrophy is a very serious and fatal disease that slowly destroys every muscle in the body. This rare disease affects one out of every 3,500 people and mostly…

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Editor’s Choice: Overcoming Obstacles and Fulfilling Dreams with Rare Disease

Happy Friday Patient Worthians! PW Contributor Tom Seaman talks about what it's like to overcome the mental toll of your rare disease diagnosis. Another PW Contributor suffering from TN, dysautonomia and…

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Duchenne Muscular Dystrophy Motivated These Parents to Start the Romito Foundation
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Duchenne Muscular Dystrophy Motivated These Parents to Start the Romito Foundation

Duchenne muscular dystrophy (DMD) affects approximately 15,000 to 20,000 young boys in the US, but for the Richard and Jamie Romito family, those numbers mean nothing. All three of their sons…

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Drug for Duchenne Muscular Dystrophy Sails Through FDA Approval, Questioned by Shareholders
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Drug for Duchenne Muscular Dystrophy Sails Through FDA Approval, Questioned by Shareholders

Duchenne muscular dystrophy (DMD) is a particularly cruel genetic disease. It most commonly affects boys, who by the age of three, begin to experience muscle weakness in the legs, hips…

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