Myotonic Dystrophy Type 1: RNA-Targeted CRISPR Shows Promise in Mouse Model
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Myotonic Dystrophy Type 1: RNA-Targeted CRISPR Shows Promise in Mouse Model

According to a story from PR Newswire, the RNA-targeted gene therapy company Locanabio, Inc. has recently released findings from a preclinical evaluation of an RNA-targeted CRISPR Cas9 (RCas9) system as…

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New Gene Editing Twist Successfully Treats Myotonic Dystrophy in Mice
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New Gene Editing Twist Successfully Treats Myotonic Dystrophy in Mice

According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…

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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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Possible New Treatment for Facioscapulohumeral Muscular Dystrophy
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Possible New Treatment for Facioscapulohumeral Muscular Dystrophy

A team of researchers from the University of Alberta have created a possible treatment for facioscapulohumeral muscular dystrophy (FSHD). Their work has been published in the Proceedings of the National Academy…

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MRFF Funding Promotes Treatment Development for Patients with Rare Diseases
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MRFF Funding Promotes Treatment Development for Patients with Rare Diseases

  When it comes to researching and finding treatments for rare diseases, funding plays a crucial role. Recently, the Medical Research Future Fund (MRFF) Stem Cell Therapies Mission presented Monash…

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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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ICYMI: SRP-9003 Study Shows Positive Results for Treating LGMD Type 2E
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ICYMI: SRP-9003 Study Shows Positive Results for Treating LGMD Type 2E

  In a recent press release, biotechnology company Sarepta Therapeutics announced positive findings from their clinical trial studying SRP-9003 as a treatment for limb-girdle muscular dystrophy (LGMD) type 2E. This…

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New Data Gives Hope for a Limb-Girdle Muscular Dystrophy Gene Therapy
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New Data Gives Hope for a Limb-Girdle Muscular Dystrophy Gene Therapy

According to a story from BioPharma Dive, the drug company Sarepta Therapeutics is pushing forward with its plans to develop a gene therapy for limb-girdle muscular dystrophy (LGMD). The company…

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Filmmaker with Muscular Dystrophy Wins Award for “Muscle Warriors” Documentary
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Filmmaker with Muscular Dystrophy Wins Award for “Muscle Warriors” Documentary

  According to Disability Horizons, an extremely talented filmmaker with muscular dystrophy recently won an Award of Recognition from The Best Shorts Film Competition. Stephanie Castelete-Tyrrell's film, entitled Muscle Warriors, documents…

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Those with Disabilities and Rare Medical Conditions in Canada are Excluded from Financial Relief During Covid-19

Kimberly Underhay Kimberly Underhay is a Canadian who has survived cancer. She has also been diagnosed with osteoarthritis and has to use a walker. Her disabilities have left her unable…

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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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This Muscular Dystrophy Patient has Spent Her Life Working to Improve the Lives of Others
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This Muscular Dystrophy Patient has Spent Her Life Working to Improve the Lives of Others

Jalee Pelissier lives with muscular dystrophy. When she was just 13 she became an advocate for others with disorders or disabilities like her own. Now 20, her advocacy has only…

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ICYMI Fulcrum Therapeutic’s Sickle Cell Disease Treatment On Track for a New Drug Application
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ICYMI Fulcrum Therapeutic’s Sickle Cell Disease Treatment On Track for a New Drug Application

As reported in Biospace, Fulcrum Therapeutics has announced it will be submitting an application to the FDA for FTX-6058 as an Investigational New Drug (IND) by the end of 2020…

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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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CRISPR as a Tool to Treat Facioscapulohumeral Muscular Dystrophy
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CRISPR as a Tool to Treat Facioscapulohumeral Muscular Dystrophy

  According to a press release from the Boston Children's Hospital, a study has discovered the efficacy of CRISPR-Cas9 gene-editing technology in the treatment of facioscapulohumeral muscular dystrophy (FSHD). As…

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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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It Just Makes “Antisense:” Using Genetic Medicine to Treat Rare Diseases
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It Just Makes “Antisense:” Using Genetic Medicine to Treat Rare Diseases

  As previously published in Scientific American, slightly after her first birthday, Emma Larson lost the use of her legs and started experiencing difficulty crawling. She was diagnosed with spinal muscular…

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Mom of Seven Year Old with Duchenne Muscular Dystrophy Speaks at Recent Festival of Genomics
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Mom of Seven Year Old with Duchenne Muscular Dystrophy Speaks at Recent Festival of Genomics

  Shelley Simmonds recently attended the Festival of Genomics, the largest genomics event in the United Kingdom that is quickly growing into the biggest event in the world. It involves…

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AskFirst: A New Collaborative Program to Support Rare Disease Patients in Gene Therapy Clinical Trials
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AskFirst: A New Collaborative Program to Support Rare Disease Patients in Gene Therapy Clinical Trials

AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina.  This company is dedicated to the research of gene therapies as potential therapeutic options…

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The Muscular Dystrophy Association Steps up for Rare Neuromuscular Diseases
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The Muscular Dystrophy Association Steps up for Rare Neuromuscular Diseases

According to a story from Charcot-Marie-Tooth News, the Muscular Dystrophy Association (MDA) may at first seem like a group that is solely focused on helping muscular dystrophy patients, but its…

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