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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For This Maroteaux-Lamy Syndrome Patient, Deportation Would Mean Certain Death
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For This Maroteaux-Lamy Syndrome Patient, Deportation Would Mean Certain Death

According to a story from Buzzfeed News, Maria Isabel Bueso was born in Guatemala with mucopolysaccharidosis VI, also known as Maroteaux-Lamy syndrome. At age seven she traveled to the US…

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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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New hATTR Amyloidosis Research Presented at Recent EU Meeting
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New hATTR Amyloidosis Research Presented at Recent EU Meeting

According to a story from BioPortfolio, the drug company Alnylam Pharmaceuticals recently presented research findings related to hATTR amyloidosis, a rare disease. The study was presented at the Second European…

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A New Approach to Halting Metastatic Cancer

A recent article in Northwestern's University Health reported that although the survival rate for cancer patients has vastly improved over the past twenty years, the deaths due to metastatic cancer…

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First Patients Recruited in Maroteaux-Lamy Syndrome Biomarker Study
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First Patients Recruited in Maroteaux-Lamy Syndrome Biomarker Study

According to a story from Benzinga, the biopharmaceutical company Inventiva has recently announced that it has recruited the first patients in a study of biomarkers in Maroteaux-Lamy syndrome. The study…

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A Proof-of-Concept Study for an Experimental Hurler Syndrome Treatment is Going Well so Far
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A Proof-of-Concept Study for an Experimental Hurler Syndrome Treatment is Going Well so Far

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Orchard Therapeutics recently announced the release of some data from an ongoing proof-of-concept study. This special trial is testing the company's…

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CRISPR Gene Editing Promises to Cure 10,000 diseases. Now in Its First Human Clinical Trial. Will it Eventually Deliver?

  Clustered Regularly Interspaced Short Palindromic Repeats is more commonly known as CRISPR. As reported recently in Science News, the CRISPR/Cas9 “molecular scissors” is slated to make its highly anticipated debut…

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FDA Begins Review of Biologics License Application for Neuromyelitis Optica Spectrum Disorder Drug
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FDA Begins Review of Biologics License Application for Neuromyelitis Optica Spectrum Disorder Drug

According to a press release from Maryland-based Viela Bio, the US Food and Drug Administration (FDA) recently agreed to review the Company's Biologics License Application for its neuromyelitis optica spectrum…

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Controlling Drug Prices Shouldn’t Harm Rare Disease Patient Access to Treatment
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Controlling Drug Prices Shouldn’t Harm Rare Disease Patient Access to Treatment

According to a story from The Globe and Mail, Canada's Patented Medicine Prices Review Board announced last month that drug prices would be subject to new regulations. These new rules…

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Biomedical Innovation Law Expert Presents Ethical Difficulties Posed by Gene Editing

According to a publication from Charcot Marie Tooth News, biomedical law expert Timo Minssen recently gave a presentation at the New York Genome Center where he discussed the potential ethical…

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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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