First Hemophilia B Patient Enrolled in Potential Breakthrough Phase III Trial

According to BioPortfolio, the company UniQure has recently announced that the first hemophilia B patient has been enrolled in their Phase III study of the treatment AMT-061, which is an…

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An Experimental Duchenne Muscular Dystrophy Gene Therapy Has Been Taken Off Clinical Hold

A Phase I/II clinical trial of the investigational gene therapy SGT-001 designed to treat Duchenne muscular dystrophy has had its clinical hold lifted by the US Food and Drug Administration…

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A Gene Therapy Being Developed to Treat Duchenne Muscular Dystrophy Has Shown Exciting Results
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A Gene Therapy Being Developed to Treat Duchenne Muscular Dystrophy Has Shown Exciting Results

An experimental gene therapy designed to treat Duchenne muscular dystrophy has produced exciting preliminary results in a Phase 1/2a clinical trial. Sarepta Therapeutics, the company behind the therapy’s development, announced…

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An Experimental Drug for Duchenne Muscular Dystrophy Has Been Awarded Orphan Drug Status by the F.D.A.
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An Experimental Drug for Duchenne Muscular Dystrophy Has Been Awarded Orphan Drug Status by the F.D.A.

The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug Sarconeos, developed by the company Biophytis, for the treatment of Duchenne muscular dystrophy. The…

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The Mother of a Boy with Duchenne Muscular Dystrophy Shares Her Family’s Experience with the Disease
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The Mother of a Boy with Duchenne Muscular Dystrophy Shares Her Family’s Experience with the Disease

William Eames, a five-year-old boy from a village in Hertfordshire, England, was diagnosed with Duchenne muscular dystrophy when he was three. Since then, his family have worked hard to raise…

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The UK Parliament Met With a Mother Advocating For Duchenne Muscular Dystrophy Patients
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The UK Parliament Met With a Mother Advocating For Duchenne Muscular Dystrophy Patients

Phillippa Farrant, whose son, Daniel, has been diagnosed with Duchenne muscular dystrophy (DMD), recently appeared in Parliament in the UK to fight for improved access to care and treatment for…

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How a Local Community Supports a Child with Duchenne Muscular Dystrophy
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How a Local Community Supports a Child with Duchenne Muscular Dystrophy

The phrase “it takes a village” gets tossed around a lot. But in Codicote, that’s what’s happening. Fundraising events and challenges are being held left and right. All of them…

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A Potential Gene Therapy for Duchenne Muscular Dystrophy Has Just Begun Patient Testing
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A Potential Gene Therapy for Duchenne Muscular Dystrophy Has Just Begun Patient Testing

Pfizer has just begun a medical trial for a potential gene therapy treatment for Duchenne muscular dystrophy, and the first patient has been dosed. Click here to read the original…

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Company Continues to Back an Experimental Therapy for Duchenne Muscular Dystrophy Despite Clinical Hold
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Company Continues to Back an Experimental Therapy for Duchenne Muscular Dystrophy Despite Clinical Hold

According to a story from genengnews.com, the pharmaceutical company Solid Biosciences is pledging to continue developing its investigation drug candidate SGT-001 for Duchenne muscular dystrophy. This is despite its clinical…

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Investigational Therapy for Duchenne Muscular Dystrophy is Seeking Orphan Drug Designation
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Investigational Therapy for Duchenne Muscular Dystrophy is Seeking Orphan Drug Designation

According to a story from financialbuzz.com, the biotechnology company BIOPHYTIS has submitted documentation for orphan drug designation from the European Medicines Agency (EMA). This designation will be for Sarconeos, a…

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Advances in Care Are Allowing Duchenne Muscular Dystrophy Patients to Live Longer, But With A Longer Life Comes New Challenges
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Advances in Care Are Allowing Duchenne Muscular Dystrophy Patients to Live Longer, But With A Longer Life Comes New Challenges

According to a story from sciencedaily.com, new and improved standards of treatment are allowing patients with Duchenne muscular dystrophy to have longer lifespans than in the past. By all accounts,…

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The FDA Puts a Halt to Trial for SGT-001, A Treatment in Development for Duchenne Muscular Dystrophy
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The FDA Puts a Halt to Trial for SGT-001, A Treatment in Development for Duchenne Muscular Dystrophy

According to a story from raredr.com, the FDA had to order the cessation of Phase II/III trials for the microdystrophin transfer therapy SGT-001, which was in development for the treatment…

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A Drug That Can Mimic Exercise May Be Closer Than You Thought
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A Drug That Can Mimic Exercise May Be Closer Than You Thought

Scientists in California at Salk Institute for Biological Studies are currently developing a drug that can mimic the effects in the body usually obtained from exercise, reports the National Post.…

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A CRISPR Gene Editing Technique Could Fix Many of the Mutations Responsible For Duchenne Muscular Dystrophy
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A CRISPR Gene Editing Technique Could Fix Many of the Mutations Responsible For Duchenne Muscular Dystrophy

CRISPR gene editing technology is still mostly in its infancy, but the new tool is already causing a lot of excitement, particularly in the treatment of genetically-linked diseases. A recent…

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The Results of this Study Could Improve Outcomes for Duchenne Muscular Dystrophy
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The Results of this Study Could Improve Outcomes for Duchenne Muscular Dystrophy

Last month, the pharmaceutical company Sarepta Therapeutics announced the long awaited results of a study that measured lung function in patients with Duchenne muscular dystrophy (DMD) after they took eteplirsen,…

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