Duchenne Muscular Dystrophy and The Heart: Five Facts You Should Know
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Duchenne Muscular Dystrophy and The Heart: Five Facts You Should Know

According to a story from blog.cincinnatichildrens.org, a diagnosis of Duchenne muscular dystrophy is always an alarming piece of news; learning that your child has a progressive muscle atrophy disorder that…

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Lithium Improves Symptoms in Mouse Model of Rare Limb Girdle Muscular Dystrophy Variant

According to a story from Science Daily, a recent study has found that lithium chloride was able to improve muscle size and strength in a mouse mode of a specific…

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Community Helps Support Ten Year Old Affected by Duchenne Muscular Dystrophy
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Community Helps Support Ten Year Old Affected by Duchenne Muscular Dystrophy

According to a story from westernmassnews.com, the community of Monson, Massachusetts is coming together to support Jason Harris and his family. Jason is ten years old and lives with Duchenne…

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Company Seeks Marketing Approval for Possible Duchenne Muscular Dystrophy Drug in EU
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Company Seeks Marketing Approval for Possible Duchenne Muscular Dystrophy Drug in EU

According to a story from BioPortfolio, the specialty pharmaceutical company Santhera Pharmaceuticals recently announced that it has submitted its marketing application to the European Medicines Agency (EMA) for its drug…

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The Design Company That Creates Custom Fashion for People With Disabilities
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The Design Company That Creates Custom Fashion for People With Disabilities

  Open Style Lab is an organization that considers itself dedicated to creating "functional wearable solutions for people of all abilities without compromising on style.” They use a wide variety…

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Program Established by the NIH to Help Rare Disease Advocacy Groups Start Patient Registries

The Beginnings Anne Pariser worked for 16 years at the FDA. For part of that time, she was a team leader for the Center for Drug Evaluation and Research where…

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New Collaboration is Dedicated to Developing a Gene Therapy for Congenital Muscular Dystrophy

According to a story from globenewswire.com, the specialty pharmaceutical company Santhera Pharmaceuticals will be entering a collaborative partnership with the University of Basel's Biozentrum. The goal of this partnership will…

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After Issues With Adverse Events, the Future of This Experimental Duchenne Muscular Dystrophy Drug Remains Unclear

According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…

Continue Reading After Issues With Adverse Events, the Future of This Experimental Duchenne Muscular Dystrophy Drug Remains Unclear

After Issues With Adverse Events, the Future of This Experimental Duchenne Muscular Dystrophy Drug Remains Unclear

According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…

Continue Reading After Issues With Adverse Events, the Future of This Experimental Duchenne Muscular Dystrophy Drug Remains Unclear

New Bill Could Allow Easier Access to Complex Rehabilitation Technology for Rare Disease Patients

H.R. 2408 H.R. 2408 is a new piece of federal legislation otherwise known as the "Ensuring Access to Quality Complex Rehabilitation Technology Act." Essentially, this act creates a new category…

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Duchenne Muscular Dystrophy Patient That Admired Stephen Hawking Passed Away During Exams…With Close to Perfect Scores
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Duchenne Muscular Dystrophy Patient That Admired Stephen Hawking Passed Away During Exams…With Close to Perfect Scores

According to a story from m.dailyhunt.in, Vinayak Sreedhar, an Indian student with Duchenne muscular dystrophy, passed away in the midst of taking his exams. Vinayak was a driven and highly…

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Researchers Believe Lithium May be Key to Developing a Drug for Limb Girdle Muscular Dystrophy

The News Hub at Washington State University recently carried an article describing initial success by researchers towards developing a drug for a rare form of muscular dystrophy. Several thousand people…

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Fulcrum Therapeutics Acquires Experimental Facioscapulohumeral Muscular Dystrophy Drug

According to a press release from Massachusetts-based biotech company Fulcrum Therapeutics, the company has successfully secured global commercialization rights of GlaxoSmithKline-developed experimental facioscapulohumeral muscular dystrophy (FSHD) drug losmapimod. Losmapimod, an…

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Muscular Dystrophy Association to Host First Annual Combined Conference
Hundreds of people living with narcolepsy gathered at the NN's 2015 Conference to make friends and learn something new. Source: Narcolepsy Network

Muscular Dystrophy Association to Host First Annual Combined Conference

A recent publication from Charcot-Marie-Tooth News announced that this year, for the first time ever, the Muscular Dystrophy Association will be hosting its annual clinical conferences at the same event…

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AMO Pharma Presents New Rating Scale for Type 1 Myotonic Dystrophy Symptoms
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AMO Pharma Presents New Rating Scale for Type 1 Myotonic Dystrophy Symptoms

A press release from American biopharmaceutical company AMO Pharma, published by PR Newswire, detailed the recent completion of the Company's new congenital myotonic dystrophy type 1 (CDM1) symptom-evaluation scale. The…

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Company Announces Plans for Myotonic Dystrophy and Duchenne Muscular Dystrophy Treatments
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Company Announces Plans for Myotonic Dystrophy and Duchenne Muscular Dystrophy Treatments

According to a story from CRWE World, the genetic medicines company Audentes Therapeutics has recently announced its intent to develop new therapies for myotonic dystrophy and Duchenne muscular dystrophy. These…

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Clinical Trial Initiated for New Technology That Could Allow Amyotrophic Lateral Sclerosis Patients to Communicate

People living with rare diseases such as amyotrophic lateral sclerosis (ALS) or muscular dystrophy as well as those who have suffered from a stroke or spinal cord injury often experience…

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Muscular Dystrophy Association Doles out $2 Million in ALS Research Grants

According to a press release from the Muscular Dystrophy Association published at Biospace, the group has resolved to award eight new research grants for the study of amyotrophic lateral sclerosis…

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Company Announces Plans to File for Conditional Marketing for Duchenne Muscular Dystrophy Drug in the EU

According to a story from Market Screener, the specialty pharmaceutical company Santhera Pharmaceuticals recently released an announcement in which the company revealed its intent to file for Conditional Marketing Authorization…

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The Struggles of being a Rare Disease Patient in China

The State of Rare Diseases in China The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this…

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Yet Another Study Shows the Dire Need for More Diversity in Medical Research

A recent study published in Cell has exhibited the scientific community's negligence for conducting research that includes an adequate representation of non-Europeans. Specifically, it showed that 78% of patients in genomic…

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First Potential Gene Therapy for Tay-Sachs Disease Produces Positive Results at Three Month Mark

Tay-Sachs disease is a rare, genetic and sadly fatal condition. It's a neurodegenerative disorder caused by an impaired production of the β-Hexosaminidase A enzyme (HEXA). Symptoms of this condition include listlessness, diminishing…

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