Investigational Therapy for Duchenne Muscular Dystrophy is Seeking Orphan Drug Designation
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Investigational Therapy for Duchenne Muscular Dystrophy is Seeking Orphan Drug Designation

According to a story from financialbuzz.com, the biotechnology company BIOPHYTIS has submitted documentation for orphan drug designation from the European Medicines Agency (EMA). This designation will be for Sarconeos, a…

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Nanotechnology Could Become a Breakthrough Treatment For Patients With Rare Cancers
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Nanotechnology Could Become a Breakthrough Treatment For Patients With Rare Cancers

According to a story from biospace.com, a research team from the National Cancer Institute was able to successfully deliver an experimental nanomedicine to mice that had rare cancers such as…

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Exposure to Diesel Exhaust Could Increase the Risk of Amyotrophic Lateral Sclerosis, Study Says
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Exposure to Diesel Exhaust Could Increase the Risk of Amyotrophic Lateral Sclerosis, Study Says

According to a story from webmd.com, diesel exhaust could increase the risk of developing amyotrophic lateral sclerosis, also known as Lou Gehrig disease. People who are exposed to diesel exhaust…

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An Experimental Treatment for Fragile X and Angelman Syndromes Gets Fast Track Designation
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An Experimental Treatment for Fragile X and Angelman Syndromes Gets Fast Track Designation

According to a story from pharmiweb.com, the pharmaceutical company Ovid Therapeutics announced recently that the U.S. Food and Drug Administration (FDA) has given its drug candidate OV101 Fast Track designation…

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A Mother and Daughter in India With Muscular Dystrophy Have Started A Hunger Strike To Demand Treatment and Support
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A Mother and Daughter in India With Muscular Dystrophy Have Started A Hunger Strike To Demand Treatment and Support

According to a story from aninews.in, mother Shashi Mishra and her daughter Anamika Mishra have staged a hunger strike in a desperate move to receive treatment for their muscular dystrophy.…

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An Apple Watch Strap is in Development That Could Detect Hyperkalemia
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An Apple Watch Strap is in Development That Could Detect Hyperkalemia

According to a story from engadget.com, the tech company AliveCor, known for its KardiaBand device for Apple Watch, is currently developing new capabilities for its innovative watch strap. Currently, the…

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Advances in Care Are Allowing Duchenne Muscular Dystrophy Patients to Live Longer, But With A Longer Life Comes New Challenges
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Advances in Care Are Allowing Duchenne Muscular Dystrophy Patients to Live Longer, But With A Longer Life Comes New Challenges

According to a story from sciencedaily.com, new and improved standards of treatment are allowing patients with Duchenne muscular dystrophy to have longer lifespans than in the past. By all accounts,…

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A Young Girl With Hydrocephalus Will Have to Travel Thousands of Miles For Treatment
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A Young Girl With Hydrocephalus Will Have to Travel Thousands of Miles For Treatment

According to a story from the Herald Sun, Matilda Mahon, an eighteen month old girl from Australia, will need to travel to the United States in order to receive treatment…

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Patient Organizations Are Starting to Risk Their Financial Ties to Big Pharma by Opposing High Drug Prices
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Patient Organizations Are Starting to Risk Their Financial Ties to Big Pharma by Opposing High Drug Prices

According to a story from Xconomy National, some patient advocacy organizations are beginning to speak out about exorbitantly high drug prices despite the fact that many of them receive funding…

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Teens and Young Adults are Less Likely to Stick to Follow-up Guidelines After Cancer Treatment
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Teens and Young Adults are Less Likely to Stick to Follow-up Guidelines After Cancer Treatment

According to a story from The European Society For Medical Oncology, a recent study shows that young adults and teenagers are less likely to adhere to follow-up guidelines following treatment…

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#PutPatientsFirst : The Trump Administration Wants to Allow Medical Personnel to Withhold Service Based on “Religious” Beliefs
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#PutPatientsFirst : The Trump Administration Wants to Allow Medical Personnel to Withhold Service Based on “Religious” Beliefs

According to a story from guttmacher.org, the U.S. Department of Health and Human Services proposed a host of new regulations purported to enforce over 20 federal provisions related to "conscience…

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These Drug Companies Are Trying to Make Treatment for Familial Hypercholestrolemia More Affordable
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These Drug Companies Are Trying to Make Treatment for Familial Hypercholestrolemia More Affordable

According to a story from PR Newswire, the pharmaceutical companies Sanofi and and Regeneron Pharmaceuticals are implementing a plan to make Praluent injections more accessible and affordable for patients with…

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Understanding The Mechanisms of Charcot-Marie-Tooth Disease Can Help Develop Treatments
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Understanding The Mechanisms of Charcot-Marie-Tooth Disease Can Help Develop Treatments

According to a story from news-medical.net, researchers from The Scripps Research Institute have successfully outlined a new path for the treatment of Charcot-Marie-Tooth disease subtype 2D (CMT2D). The scientists hope…

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A Treatment for Batten Disease Was Given Rare Pediatric Disease Designation from the FDA
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A Treatment for Batten Disease Was Given Rare Pediatric Disease Designation from the FDA

According to a story from pharmpro.com, the pharmaceutical company Abeona Therapeutics announced that it has been given Rare Pediatric Disease Designation for its ABO-202 program by the U.S. Food and…

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The FDA Gave Orphan Drug Designation to an Investigational Treatment For Cystic Fibrosis
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The FDA Gave Orphan Drug Designation to an Investigational Treatment For Cystic Fibrosis

According to a story from Financial Buzz, the pharmaceutical company Proteostasis Therapeutics recently announced that the company's experimental treatment PTI-428 was granted Orphan Drug Designation by the FDA. PTI-428 is…

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