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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HOPE-2 Clinical Trial for Duchenne Muscular Dystrophy is on Track to Resume

What is Duchenne Muscular Dystrophy? Duchenne muscular dystrophy (DMD) is a rare genetic condition that is ultimately fatal. It causes muscles to weaken and usually results in heart failure before age…

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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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Company Provides Updates for Phase 3 Duchenne Muscular Dystrophy Trial
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Company Provides Updates for Phase 3 Duchenne Muscular Dystrophy Trial

According to a story from drugs.com, the biopharmaceutical company Catabasis pharmaceuticals recently provided updates on the status of its Phase 3 clinical trial testing its investigational therapy CAT-1004, also known…

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Potential Gene Therapy for Limb Girdle Muscular Dystrophy Type 2D Granted Orphan Drug Designation

Limb Girdle Muscular Dystrophy Type 2D (LGMD2D), also called alpha-sarcoglycanopathy, is a form of muscular dystrophy which is caused by a defected SGCA gene. It causes the body to lose function of…

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ICYMI: Experimental Treatment for Duchenne Muscular Dystrophy Shows Potential in Early Trial
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ICYMI: Experimental Treatment for Duchenne Muscular Dystrophy Shows Potential in Early Trial

According to a story from globenewswire.com, the biotechnology company Wave Life Sciences Ltd. recently announced that the tolerability and safety data from its Phase 1 clinical trial of its investigational therapy…

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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients
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Device Allows Monitoring of Lung Function at Home For Duchenne Muscular Dystrophy Patients

According to a story from EurekAlert!, monitoring lung function is an important role of managing Duchenne muscular dystrophy and other diseases that can cause wasting of the respiratory muscles. Many…

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Man With Duchenne Muscular Dystrophy Celebrates 50th Birthday After Grim Prognosis as a Child
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Man With Duchenne Muscular Dystrophy Celebrates 50th Birthday After Grim Prognosis as a Child

  Andrew Taylor celebrated his 50th birthday this year. This is a big year for anyone, but especially for Taylor, who was told he would not make it to the…

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34 Years After Bhopal Disaster, Chronic Health Conditions Persist
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34 Years After Bhopal Disaster, Chronic Health Conditions Persist

Earlier this month marked a solemn 34 years since the horrendous Bhopal disaster. The worst industrial disaster in human history killed over 3,800 almost instantly, and tens of thousands in…

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