Kevin Hart Gives a Hand to the Muscular Dystrophy Association As Host of the MDA Telethon
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Kevin Hart Gives a Hand to the Muscular Dystrophy Association As Host of the MDA Telethon

As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…

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Editor’s Choice: Balance Awareness Week and Study Updates

Happy Friday! Sept. 13-19 is Balance Awareness Week. It's intended to bring attention to balance-related vestibular disorders such as Meniere’s disease and vestibular migraine. Looking for ways to help out?…

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FDA Accepts Casimersen NDA for Patients with Exon 45 Amenable DMD

Earlier this week, Sarepta Therapeutics announced that the FDA approved a New Drug Application (NDA) for Casimersen (SRP-4045). The treatment, for patients with exon 45 amenable Duchenne muscular dystrophy (DMD),…

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Fast Track Designation Granted for Gene Therapy Treatment of Duchenne Muscular Dystrophy
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Fast Track Designation Granted for Gene Therapy Treatment of Duchenne Muscular Dystrophy

by Lauren Taylor from In The Cloud Copy Duchenne muscular dystrophy (DMD) is a rare genetic condition that ultimately leads to the wasting away of muscles over time. The condition…

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Enrollment Begins in Trial of Duchenne Muscular Dystrophy Treatment

According to BioSpace, the first patient has been enrolled in a trial of pamrevlumab, a Duchenne muscular dystrophy treatment. The third phase of LELANTOS plans to evaluate the drug's effects…

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Researchers Produce End-to-End X Chromosome DNA sequence

  As society becomes increasingly technological, researchers are getting closer and closer to mapping the entire human genome. In facts, shares Medical XPress, we may be nearly there. While many…

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Duchenne Muscular Dystrophy: Two Patients with a Rare Mutation Were Just Dosed with Gene Therapy
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Duchenne Muscular Dystrophy: Two Patients with a Rare Mutation Were Just Dosed with Gene Therapy

According to a story from the CureDuchenne Blog, Dr. Kevin Flanigan of Nationwide Children's Hospital has recently dosed two Duchenne muscular dystrophy patients with an experimental gene therapy. This gene…

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Phase 2 Trial Announced for Potential COVID-19 Treatment TXA127
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Phase 2 Trial Announced for Potential COVID-19 Treatment TXA127

  As COVID-19 continues to spread, the rush for a treatment or cure continues. Today, on August 4, there are 18.2 million diagnosed cases worldwide, with an associated 692,000 deaths.…

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Recruitment for South Korea’s Rare Disease National Bio Big Data Project Begins
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Recruitment for South Korea’s Rare Disease National Bio Big Data Project Begins

According to a story from Korea Biomedical Review, Seoul National University Hospital (SNUH) in South Korea has just announced recently that the process of recruiting patients for the National Bio…

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Study: Gene Therapy as a Treatment for Duchenne Muscular Dystrophy
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Study: Gene Therapy as a Treatment for Duchenne Muscular Dystrophy

According to Medical Xpress, a new gene therapy has been studied for the treatment of Duchenne muscular dystrophy. This treatment, SRP-9001, has been shown to deliver micro-dystrophin and improve functional…

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A New Partnership will Advance Exosome-Based Gene Therapy for Neuromuscular Diseases
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A New Partnership will Advance Exosome-Based Gene Therapy for Neuromuscular Diseases

By engineering exosomes, Sarepta Therapeutics believes they can utilize gene therapies, RNA therapies, and gene editing without triggering an immune response in the body. They have just announced a new…

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New Pediatric Certified Duchenne Muscular Dystrophy Care Center in Texas
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New Pediatric Certified Duchenne Muscular Dystrophy Care Center in Texas

Parent Project Muscular Dystrophy is a nonprofit that aims to find a cure for Duchenne muscular dystrophy. One of their programs, the Certified Duchenne Care Center Program (CDCCP), has recently…

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How Rare Disease Biotech “Cure Rare Disease” Is Handling COVID-19
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How Rare Disease Biotech “Cure Rare Disease” Is Handling COVID-19

From social distancing and shuttered businesses to personal health concerns, COVID-19 has changed daily life for the foreseeable future. There are nearly 4.75 million diagnoses worldwide, with 1.53 million in…

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COVID-19 Advice for Patients with Becker or Duchenne Muscular Dystrophy

With 4.15 million cases across the globe, COVID-19 is at the forefront of discussion. Knowledge about the viral pandemic continues to grow each and every day. Yet there are still…

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CureDuchenne Announces Partnership with Myosana Therapeutics for Duchenne Muscular Dystrophy Treatment
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CureDuchenne Announces Partnership with Myosana Therapeutics for Duchenne Muscular Dystrophy Treatment

According to a story from the CureDuchenne Blog, CureDuchenne founder and CEO Debra Miller announced that the organization has recently entered into a new partnership with the biotechnology company Myosana…

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Boston Man Tackling Duchenne Muscular Dystrophy Cure After Sibling’s Diagnosis
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Boston Man Tackling Duchenne Muscular Dystrophy Cure After Sibling’s Diagnosis

by Jodee Redmond from In The Cloud Copy Rich Horgan is the founder of the Boston-based biotech non-profit, Cure Rare Diseases. The 28-year-old decided to take action over concerns about…

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Patient Involvement in Genomic Research: Unpacking the “Genetics Unzipped” Podcast Episode S3.08

  While genomic research presents the unique opportunity of combating diseases and gene malfunctions at the source, the practice has been incredibly controversial. Some people worry about the ethics of…

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Study Discovers New Method to Predict Duchenne Muscular Dystrophy Progression
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Study Discovers New Method to Predict Duchenne Muscular Dystrophy Progression

According to a study published in the journal Neurology, a measure called the vastus lateralis fat fraction was found to be useful in predicting the age at which a patient living…

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Company Starts Compassionate Use Program for an Experimental COVID-19 Treatment
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Company Starts Compassionate Use Program for an Experimental COVID-19 Treatment

According to a story from gurufocus.com, the biotechnology company Capricor Therapeutics has recently announced the initiation of a compassionate use program for its experimental drug CAP-1002. This drug will be…

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New Equity Investment Aims to Develop New Duchenne Muscular Dystrophy Drug
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New Equity Investment Aims to Develop New Duchenne Muscular Dystrophy Drug

According to a story from CureDuchenne, the biotechnology company Dyne Therapeutics recently announced that CureDuchenne Ventures has made an equity investment that will support the development of all new precision…

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