A Patient Registry is Critical for Advancing Myalgic Encephalomyelitis Research
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A Patient Registry is Critical for Advancing Myalgic Encephalomyelitis Research

According to a story from meaction.net, Jaime Seltzer, the Director of Scientific and Medical Outreach at #MEAction, recently spoke at the Third Annual Working Group Meeting on the Molecular Basis…

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An Experimental Treatment for Pulmonary Arterial Hypertension Just Earned Orphan Drug Designation

According to a story from firstworldpharma.com, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that the company's experimental product candidate sotatercept has earned Orphan Drug designation from the US Food…

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Companies Reach Partnership Agreement Related to an Experimental Treatment for ATTR Amyloidosis
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Companies Reach Partnership Agreement Related to an Experimental Treatment for ATTR Amyloidosis

According to a story from BioPortfolio, Alexion Pharmaceuticals, Inc. and Eidos Therapeutics, Inc., have recently struck an agreement related to the development and marketing of an experimental treatment for hATTR…

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Making History: First Drug Approved for Scleroderma-Associated Interstitial Lung Disease
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Making History: First Drug Approved for Scleroderma-Associated Interstitial Lung Disease

According to a story from finanzen.at, the drug nintedanib (marketed as Ofev) has officially been approved by the US Food and Drug Administration (FDA) as a treatment for interstitial lung…

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Chronic Kidney Disease is Just One of Many Diseases That Will Become More Prevalent Because of Climate Change
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Chronic Kidney Disease is Just One of Many Diseases That Will Become More Prevalent Because of Climate Change

According to a story from EurekAlert!, farm workers from across the world are experiencing increased rates of chronic kidney disease. The precise reason for the increased rates are unknown, but…

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Attention Tyrosinemia Community Families: Come Together for Event in Late September

September is Tyrosinemia Awareness Month! The good people of the Network Of  Tyrosinemia Advocates (N.O.T.A.) have organized a family weekend later this month aimed to bring the Tyrosinemia community together. Let's…

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Biohacking: A Small Group of Amateur Biologists Are Hoping to Market Their Knockoff of a One Million Dollar Gene Therapy

  According to a recent article in Archive Today, an international band of amateur biologists, or "biohackers" announced that they intend to eventually market a knock-off of Glybera, a one…

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The Power of Collective Action in Providing Insurance Coverage for Spinal Muscular Atrophy Gene Therapy

Maisie Green Maisie Green is a 20-month-old little girl whose insurance has finally approved coverage for a life-altering spinal muscular atrophy treatment. Two weeks ago she received the singular infusion…

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Zogenix Acquires Modis in $400 Million Buyout, Adds Experimental TK2 mDNA Depletion Syndrome Treatment to Pipeline

According to a press release from California-based biotechnology company Zogenix, the Company recently completed a multi-million dollar acquisition of Modis Therapeutics. Before its acquisition, Modis focused on the development of…

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Patient Advocacy Groups Call for Indian Health Ministry to Implement Interim Care Policy for Rare Disease Patients
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Patient Advocacy Groups Call for Indian Health Ministry to Implement Interim Care Policy for Rare Disease Patients

According to a recent publication from Moneycontrol, patient advocacy groups in India are calling for the country's Minister of Health and Family Welfare, Harsh Vardhan, to establish a provisional system…

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Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome
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Korean Company Predicts Your Risk of Developing Certain Genetic Conditions by Sequencing Your Genome

According to a publication from the Korea Times, the Eone Diagnomics Genome Center in Incheon, South Korea, is working to improve patients' accessibility to their own genetic sequence. Researchers believe…

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FDA Grants Regenerative Medicine Advanced Therapy Designation to Investigational X-Linked SCID Therapy
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FDA Grants Regenerative Medicine Advanced Therapy Designation to Investigational X-Linked SCID Therapy

According to a press release from American biotechnology company Fortress Biotech, the American Food and Drug Administration (FDA) recently awarded Regenerative Medicine Advanced Therapy designation to the experimental gene therapy…

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PointR Data Merges into Mateon Therapeutics — Company Will Focus on Using Data Analysis and AI to Develop New Treatments
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PointR Data Merges into Mateon Therapeutics — Company Will Focus on Using Data Analysis and AI to Develop New Treatments

According to a press release from Mateon Therapeutics, Mateon's recent merger agreement with PointR Data establishes plans to combine the use of artificial intelligence and data collection for the development…

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Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation
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Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company CrystalGenomics, Inc. recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational drug candidate CG-745 Orphan…

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