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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Losmapimod Receives Orphan Drug Designation for the Treatment of FSHD
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Losmapimod Receives Orphan Drug Designation for the Treatment of FSHD

Losmapimod is an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD), and it recently received the Orphan Drug designation from the FDA. Not only did it receive this designation, but its…

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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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Translarna Results in a Preserved Ability to Walk in Those with Duchenne Muscular Dystrophy
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Translarna Results in a Preserved Ability to Walk in Those with Duchenne Muscular Dystrophy

PTC Therapeutics is a pharmaceutical company that has been working with Translarna, a medication intended for the treatment of Duchenne muscular dystrophy (DMD). They analyzed this treatment and presented the…

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As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action
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As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action

According to a story from newsroom.heart.org, one of the most substantial domestic policy achievements of the Obama administration was the passage of the Affordable Care Act (ACA). Since its passage,…

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The Muscular Dystrophy Association Awards Over $1M Towards Friedreich’s Ataxia Gene Therapy
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The Muscular Dystrophy Association Awards Over $1M Towards Friedreich’s Ataxia Gene Therapy

According to an announcement from the Muscular Dystrophy Association, the organization has recently awarded a total of $1,076,232 in MDA Venture Philanthropy (MVP) funding towards the drug company AavantiBio, which…

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Treatment for Duchenne Muscular Dystrophy with Rare Mutations Accepted Under Accelerated Approval
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Treatment for Duchenne Muscular Dystrophy with Rare Mutations Accepted Under Accelerated Approval

According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved the drug golodirsen (marketed as Vyondys 53) as a treatment for Duchenne muscular dystrophy…

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A New Pediatric Precision Medicine Center is Opening in Utah
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A New Pediatric Precision Medicine Center is Opening in Utah

A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…

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Muscular Dystrophy Association’s Pompe Disease Patient Focused Drug Development Meeting Cleared by FDA
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Muscular Dystrophy Association’s Pompe Disease Patient Focused Drug Development Meeting Cleared by FDA

According to a story from tradeshownews.vporoom.com, the Muscular Dystrophy Association (MDA) has recently announced the official approval of its Patient-Focused Drug Development (PFDD) meeting by the US Food and Drug…

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Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes
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Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes

According to a story from PR Newswire, the nonprofit groups Duchenne UK and Parent Project Muscular Dystrophy (PPMD) have jointly awarded grant funding to the tune of $200,000 to Dr.…

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Experimental Facioscapulohumeral Dystrophy Drug Found Safe and Tolerable in Phase 1 Study
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Experimental Facioscapulohumeral Dystrophy Drug Found Safe and Tolerable in Phase 1 Study

A recent press release from Fulcrum Therapeutics, a Massachusetts-based biotechnology company, announced preliminary findings of the company's phase 1 clinical study of losmapimod in the treatment of facioscapulohumeral dystrophy. Losmapimod is…

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Study Analyzes New Outcome Measure for Duchenne Muscular Dystrophy
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Study Analyzes New Outcome Measure for Duchenne Muscular Dystrophy

According to a story from BioSpace, a global collaborative called the Trajectory Analysis Project recently released a study that utilized the North Star Ambulatory Assessment (NSAA), a recently developed measurement…

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Experimental Treatment for Facioscapulohumeral Muscular Dystrophy has Disappointing Results but May Still Benefit Charcot-Marie-Tooth Disease

The Bad News Unfortunately, it has recently been announced that an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD) will not be continuing on in clinical trials. This is because the…

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Steadman Philippon Research Institute Receives Award for Duchenne Muscular Dystrophy Research

According to a story from Financial Buzz, the Steadman Philippon Research Institute (SPRI) recently received an award in research from the National Institutes of Health (NIH). The award is in…

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NeuroGen to Host No-Charge Outpatient Stem Cell Therapy Consultation Camp in Chandigarh
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NeuroGen to Host No-Charge Outpatient Stem Cell Therapy Consultation Camp in Chandigarh

According to a publication from NewZNew, the NeuroGen Brain and Spine Institute in India plans on hosting an outpatient stem cell therapy consultation camp in Chandigarh on the last day…

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Advocates for Duchenne Muscular Dystrophy Start Company Designed to Collect Data Directly From Patients
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Advocates for Duchenne Muscular Dystrophy Start Company Designed to Collect Data Directly From Patients

According to a story from Xconomy, the US Food and Drug Administration (FDA) made a controversial decision three years ago when it approved the drug eteplirsen as a treatment for…

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Early Data Released From Phase 1B Duchenne Muscular Dystrophy Gene Therapy Trial
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Early Data Released From Phase 1B Duchenne Muscular Dystrophy Gene Therapy Trial

According to a story from BioPortfolio, the drug company Pfizer has recently presented data from its phase 1b clinical trial that tested the company's experimental gene therapy PF-06939926, which is…

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