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 Treatment for Muscular Dystrophy Also Works for a Rare Heart Condition
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A Treatment for Muscular Dystrophy Also Works for a Rare Heart Condition

Researchers at the University of Hong Kong have discovered that a drug used to treat muscular dystrophy also works on a hereditary heart disease called Lamin A/C-related dilated cardiomyopathy. The…

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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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Potential Muscular Dystrophy Treatment Faces Safety Concerns and Lawsuits
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Potential Muscular Dystrophy Treatment Faces Safety Concerns and Lawsuits

Clinical trials of a potential gene therapy treatment for Duchenne muscular dystrophy (DMD) have been put on hold after concerns emerged about the safety of the procedure, reports Genetic Engineering…

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Active Man Sets an Example of The Success of Mechanical Ventilation Technology
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Active Man Sets an Example of The Success of Mechanical Ventilation Technology

According to a story from Business Wire, there was a time when having to use a mechanical ventilator in order to breathe meant being confined at the hospital in an…

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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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New Treatments Slow The Progression of Muscular Dystrophy, Study Says
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New Treatments Slow The Progression of Muscular Dystrophy, Study Says

According to a story from Medical Xpress, two new treatments were able to slow down the worsening of symptoms associated with muscular dystrophy in a mouse model study. Tamoxifen and…

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A Mother and Daughter in India With Muscular Dystrophy Have Started A Hunger Strike To Demand Treatment and Support
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A Mother and Daughter in India With Muscular Dystrophy Have Started A Hunger Strike To Demand Treatment and Support

According to a story from aninews.in, mother Shashi Mishra and her daughter Anamika Mishra have staged a hunger strike in a desperate move to receive treatment for their muscular dystrophy.…

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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 is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials
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The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials

According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…

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Event Update: Patient-Focused Duchenne Muscular Dystrophy Meeting
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Event Update: Patient-Focused Duchenne Muscular Dystrophy Meeting

On March 5th, Parent Project Muscular Dystrophy hosted a new kind of meeting at their annual conference. Parent Project Muscular Dystrophy (PPMD) held the meeting in Washington DC with a…

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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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A.I. Bodysuit May Revolutionize Duchenne Muscular Dystrophy treatment
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A.I. Bodysuit May Revolutionize Duchenne Muscular Dystrophy treatment

The Imperial College of London recently began development of a body suit to improve treatment of Duchenne muscular dystrophy. The suit uses a unique artificial intelligence interface to collect and…

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Gene Therapy Could Be a New Treatment for Muscular Dystrophy
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Gene Therapy Could Be a New Treatment for Muscular Dystrophy

The biotech company Myonexus Therapeutics may soon begin clinical trials for muscular dystrophy gene therapy treatment after obtaining nearly $2.5 million of investments. The research behind the new treatment was…

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Spinraza, a New Drug For SMA Patients Might Be Available Sooner Than Later in the UK
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Spinraza, a New Drug For SMA Patients Might Be Available Sooner Than Later in the UK

There is new hope for patients and their families suffering from spinal muscular atrophy (SMA) reported AOL UK News. SMA is a physically debilitating and aggressive disease, and now a new…

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Researchers Close to Medical Breakthroughs Concerning  Amyotrophic Lateral Sclerosis and Muscular Dystrophy
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Researchers Close to Medical Breakthroughs Concerning Amyotrophic Lateral Sclerosis and Muscular Dystrophy

Originally reported by Columbia Daily Tribune, researchers from the University of Missouri have made huge strides towards treating, and maybe even curing, two fatal diseases: muscular dystrophy and Amyotrophic Lateral…

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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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First Duchenne Muscular Dystrophy Patient treated With New Gene Therapy

On January 17th, Parent Project Muscular Dystrophy announced that the first patient had been treated with a new gene therapy. The therapy developed specifically for Duchenne muscular dystrophy is known as…

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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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