CRISPR Successfully Treats Duchenne Muscular Dystrophy in Mouse Model
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CRISPR Successfully Treats Duchenne Muscular Dystrophy in Mouse Model

According to a story from Fierce Biotech, the results of a year-long study examining the utility of the groundbreaking gene-editing technology CRISPR in treating the rare genetic disorder Duchenne muscular…

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Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects
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Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects

Passsage Bio Passage Bio has just announced that they've received 115.5 million dollars to help advance their development of gene therapies. They will begin by using the funds to further…

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New Funding Program to Support Newborn Screening for Duchenne Muscular Dystrophy
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New Funding Program to Support Newborn Screening for Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a rare, progressive, and fatal condition. It primarily affects males (1 out of every 3,600 to 6,000 male births). It's caused by…

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HOPE-2 Clinical Trial for Duchenne Muscular Dystrophy is on Track to Resume

What is Duchenne Muscular Dystrophy? Duchenne muscular dystrophy (DMD) is a rare genetic condition that is ultimately fatal. It causes muscles to weaken and usually results in heart failure before age…

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Company Releases Early Data and Plans to Boost Dose in Duchenne Muscular Dystrophy Trial
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Company Releases Early Data and Plans to Boost Dose in Duchenne Muscular Dystrophy Trial

According to a story from pm360online.com, the life sciences company Solid Biosciences has released some preliminary findings from its Phase 1/2 clinical trial of SGT-001. This investigational product is in…

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You Can Provide Suggestions for the FDA’s Revision of their Rare Disease Research Guidelines

Time for Revision Like many things in this world, research for rare diseases such as Spinal Muscular Atrophy (SMA), Multiple Sclerosis, and Duchenne Muscular Dystrophy (DMD) has evolved substantially in recent years. This evolution…

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Company Provides Updates for Phase 3 Duchenne Muscular Dystrophy Trial
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Company Provides Updates for Phase 3 Duchenne Muscular Dystrophy Trial

According to a story from drugs.com, the biopharmaceutical company Catabasis pharmaceuticals recently provided updates on the status of its Phase 3 clinical trial testing its investigational therapy CAT-1004, also known…

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Potential Gene Therapy for Limb Girdle Muscular Dystrophy Type 2D Granted Orphan Drug Designation

Limb Girdle Muscular Dystrophy Type 2D (LGMD2D), also called alpha-sarcoglycanopathy, is a form of muscular dystrophy which is caused by a defected SGCA gene. It causes the body to lose function of…

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ICYMI: Experimental Treatment for Duchenne Muscular Dystrophy Shows Potential in Early Trial
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ICYMI: Experimental Treatment for Duchenne Muscular Dystrophy Shows Potential in Early Trial

According to a story from globenewswire.com, the biotechnology company Wave Life Sciences Ltd. recently announced that the tolerability and safety data from its Phase 1 clinical trial of its investigational therapy…

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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients
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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients

According to a story from EurekAlert!, monitoring lung function is an important role of managing Duchenne muscular dystrophy and other diseases that can cause wasting of the respiratory muscles. Many…

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Man With Duchenne Muscular Dystrophy Celebrates 50th Birthday After Grim Prognosis as a Child
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Man With Duchenne Muscular Dystrophy Celebrates 50th Birthday After Grim Prognosis as a Child

  Andrew Taylor celebrated his 50th birthday this year. This is a big year for anyone, but especially for Taylor, who was told he would not make it to the…

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34 Years After Bhopal Disaster, Chronic Health Conditions Persist
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34 Years After Bhopal Disaster, Chronic Health Conditions Persist

Earlier this month marked a solemn 34 years since the horrendous Bhopal disaster. The worst industrial disaster in human history killed over 3,800 almost instantly, and tens of thousands in…

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This Just In: India Never Implemented their Rare Disease Policy as Promised
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This Just In: India Never Implemented their Rare Disease Policy as Promised

We all got really excited last year when India implemented a new plan to benefit rare disease patients. It was called the National Rare Disease Policy. Basically, it put 12.86…

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A Clinical Trial for Gene Therapy is Underway for Muscular Dystrophy!
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A Clinical Trial for Gene Therapy is Underway for Muscular Dystrophy!

Limb-Girdle Muscular Dystrophy or LGMD, is a form of Muscular Dystrophy caused by a mutation which disrupts production of the protein beta-sarcoglycan. There are currently no treatment options for LGMD and…

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Study Shows Importance of Biobanks For Rare Disease Research
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Study Shows Importance of Biobanks For Rare Disease Research

Biobanks store biological samples to help scientists research certain diseases. Registries collect information from patients and standardize it, but no samples are stored. Which of these is best for rare…

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Researchers were Wrong about the Cause of Muscular Dystrophy
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Researchers were Wrong about the Cause of Muscular Dystrophy

For a long time, neuromuscular diseases such as muscular dystrophy, ALS, and inclusion body myopathy were thought to be caused as a result of toxic clumps of proteins such as TDP-43.…

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