Muscular Dystrophy

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

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…

Continue Reading Experimental Treatment for Facioscapulohumeral Muscular Dystrophy has Disappointing Results but May Still Benefit Charcot-Marie-Tooth Disease
Steadman Philippon Research Institute Receives Award for Duchenne Muscular Dystrophy Research
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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…

Continue Reading Steadman Philippon Research Institute Receives Award for Duchenne Muscular Dystrophy Research
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…

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

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

Continue Reading Duchenne Muscular Dystrophy and The Heart: Five Facts You Should Know
Lithium Improves Symptoms in Mouse Model of Rare Limb Girdle Muscular Dystrophy Variant
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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…

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

Continue Reading Community Helps Support Ten Year Old Affected by Duchenne Muscular Dystrophy
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…

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

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
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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…

Continue Reading New Collaboration is Dedicated to Developing a Gene Therapy for Congenital Muscular Dystrophy
After Issues With Adverse Events, the Future of This Experimental Duchenne Muscular Dystrophy Drug Remains Unclear
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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
Parentingupstream / Pixabay

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

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…

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

Continue Reading Duchenne Muscular Dystrophy Patient That Admired Stephen Hawking Passed Away During Exams…With Close to Perfect Scores
Researchers Believe Lithium May be Key to Developing a Drug for Limb Girdle Muscular Dystrophy

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…

Continue Reading Researchers Believe Lithium May be Key to Developing a Drug for Limb Girdle Muscular Dystrophy
Fulcrum Therapeutics Acquires Experimental Facioscapulohumeral Muscular Dystrophy Drug

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…

Continue Reading Fulcrum Therapeutics Acquires Experimental Facioscapulohumeral Muscular Dystrophy Drug
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…

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

Continue Reading Company Announces Plans for Myotonic Dystrophy and Duchenne Muscular Dystrophy Treatments
Clinical Trial Initiated for New Technology That Could Allow Amyotrophic Lateral Sclerosis Patients to Communicate

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…

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