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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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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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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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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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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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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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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This Journal Highlights Current Research on Neuromuscular Diseases (NMD)

In a Special Issue of the journal Disease Models and Mechanisms Highlights, James J. Dowling et al describes pediatric neuromuscular diseases as mostly genetic and affecting areas of the peripheral…

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