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

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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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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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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Faulty Proteins and Failed Genes: Understanding the Biology Behind Congenital Diseases
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Faulty Proteins and Failed Genes: Understanding the Biology Behind Congenital Diseases

Patients with congenital diseases often experience a range of uncomfortable symptoms. On one side of the world, a family with erythromelalgia, a painful condition which can cause redness and swelling…

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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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Neurodegenerative-Neuromuscular Diseases and Airway Clearance Therapy
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Neurodegenerative-Neuromuscular Diseases and Airway Clearance Therapy

  Advances in medicine have been responsible for longer overall survival in patients with neurodegenerative-neuromuscular diseases. According to a recent report in RTMagazine, an increase in the number of patients…

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The FDA is Developing, Approving, and Expediting Exciting New Therapies
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The FDA is Developing, Approving, and Expediting Exciting New Therapies

  Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a…

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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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Does the ‘Quality Adjusted Life Year’ Measure Leave Behind Rare Disease Patients?
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Does the ‘Quality Adjusted Life Year’ Measure Leave Behind Rare Disease Patients?

According to a story from Salon, the Quality Adjusted Life Year (QALY) is a statistical measure that is used in order to calculate the value of a certain medication. First…

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With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…

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