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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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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The Cure Rare Disease Foundation is Trying to Change the “One Size Fits All” Approach Toward Treatment

The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…

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Could Stopping Using the Word “Rare” Increase the Rate of Diagnosis for Rare Disease Patients?

The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…

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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
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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Rare Diseases: Only 10% of India’s 1.37 Billion People Have Health Insurance

In about five years India’s population will exceed that of China which is currently ranked number one according to population. Contrast these numbers with the fact that only ten percent…

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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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Orphan Drug Designation Granted for Potential Duchenne Muscular Dystrophy Treatment

For years FibroGen has been investigating a drug called pamrevlumab. They believe it could be an effective treatment for Duchenne muscular dystrophy (DMD), pancreatic cancer, and idiopathic pulmonary fibrosis (IPF).…

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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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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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Bioscience Companies in Ohio Take Aim at Rare Diseases
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Bioscience Companies in Ohio Take Aim at Rare Diseases

According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…

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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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Online Gaming Allowed Duchenne Muscular Dystrophy Patient to Transcend Physical Limits
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Online Gaming Allowed Duchenne Muscular Dystrophy Patient to Transcend Physical Limits

According to a story from BBC, Mats Steen passed away at 25 years old due to complications from his Duchenne muscular dystrophy. Upon his death, parents Robert and Trude grieved…

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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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