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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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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CRISPR Successfully Treats Duchenne Muscular Dystrophy in Mouse Model
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CRISPR Successfully Treats Duchenne Muscular Dystrophy in Mouse Model

According to a story from Fierce Biotech, the results of a year-long study examining the utility of the groundbreaking gene-editing technology CRISPR in treating the rare genetic disorder Duchenne muscular…

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Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects
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Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects

Passsage Bio Passage Bio has just announced that they've received 115.5 million dollars to help advance their development of gene therapies. They will begin by using the funds to further…

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New Funding Program to Support Newborn Screening for Duchenne Muscular Dystrophy
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New Funding Program to Support Newborn Screening for Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a rare, progressive, and fatal condition. It primarily affects males (1 out of every 3,600 to 6,000 male births). It's caused by…

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Company Releases Early Data and Plans to Boost Dose in Duchenne Muscular Dystrophy Trial
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Company Releases Early Data and Plans to Boost Dose in Duchenne Muscular Dystrophy Trial

According to a story from pm360online.com, the life sciences company Solid Biosciences has released some preliminary findings from its Phase 1/2 clinical trial of SGT-001. This investigational product is in…

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You Can Provide Suggestions for the FDA’s Revision of their Rare Disease Research Guidelines

Time for Revision Like many things in this world, research for rare diseases such as Spinal Muscular Atrophy (SMA), Multiple Sclerosis, and Duchenne Muscular Dystrophy (DMD) has evolved substantially in recent years. This evolution…

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