Extremely Positive Results Announced from Interim Analysis of Phase 2 Clinical Trial for Hemochromatosis

Hereditary Hemochromatosis Hereditary hemochromatosis (HH) is a rare disease caused by hepcidin deficiency or hepcidin insensitivity. Hepcidin naturally regulates iron absorption/distribution in the body. Without hepcidin, HH patients suffer from…

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Very First Patient Dosed with Investigational Therapy for Friedreich’s Ataxia in Phase 2 Clinical Trial

Friedreich's Ataxia Friedreich's ataxia (FRDA) is a rare disease which is caused by a frataxin deficiency. This deficiency results in the degeneration of nerves within the spinal cord. This nerve…

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“I Am My Own Best Asset”: Learning to Be Your Own Advocate When Living with a Rare Disease or Chronic Illness

The Danger of Stereotypes There is still a huge stereotype that women are prone to "hysteria." This leads to a general distrust in women's self-reported symptoms. Physicians consciously or unconsciously…

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Experimental Treatment for Spinocerebellar Ataxia Earns Orphan Drug Designation from the FDA

According to a story from Business Wire, the drug developer Cadent Therapeutics recently announced that its investigational drug CAD-1883 has been awarded Orphan Drug Designation from the US Food and…

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NuEyes: Using Virtual Reality to Improve Vision for Achromatopsia, Congenital Nystagmus & Macular Degeneration Patients

An Idea Sometimes life leads us down unexpected paths. Mark Greget founded a medical device distribution company. That was his plan. But after interacting with patients through his work, he…

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Study Links IL-1 Cytokines to Increased Scarring in Systemic Scleroderma Patients

According to a publication from Scleroderma News, a study recently published in Clinical & Transitional Immunology suggests that certain cytokines (small proteins that play an important role in communication between…

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Orchard Therapeutics Acquires Global Licensing Agreement for Experimental Mucopolysaccharidosis Drug
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Orchard Therapeutics Acquires Global Licensing Agreement for Experimental Mucopolysaccharidosis Drug

According to a publication from Biospace, British clinical drug developer Orchard Therapeutics has secured global intellectual property rights to research, manufacture, and market a gene therapy program for the treatment…

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The Challenge: He is A Father, A Caregiver and A Scientist Trying to Save his Son and Others With ME/CFS or Chronic Fatigue Syndrome

  Ron Davis and his gene-sequencing technologies were mentioned years ago in The Atlantic alongside Elon Musk (SpaceX) and Jeff Bezos of Amazon fame. Since then, Davis has amassed over…

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NIH Grants Funding for Scientist’s Heparin-Induced Thrombocytopenia Biomarker Research
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NIH Grants Funding for Scientist’s Heparin-Induced Thrombocytopenia Biomarker Research

According to a publication on EurekAlert, the National Institutes of Health recently awarded Dr. Jason Karnes, PharmD, PhD (and assistant professor in the University of Arizona College of Pharmacy) with…

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Could Blood Donations Improve Diagnostic Rates for Familial Hypercholesterolemia?
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Could Blood Donations Improve Diagnostic Rates for Familial Hypercholesterolemia?

According to a story from brightsurf.com, familial hypercholesterolemia, a genetic condition that causes elevated levels of LDL cholesterol to appear at a young age, often goes undiagnosed. In fact, some…

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Patient Registry Reveals Critical Information About Idiopathic Pulmonary Fibrosis

According to a story from blogs.biomedcentral.com, the findings from the IPF-PRO Registry, which is a US-based registry for idiopathic pulmonary fibrosis patients, were recently published. Patient registries are a critical…

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New Collaboration Will Test Live Biotherapeutics Against Different Cancers
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New Collaboration Will Test Live Biotherapeutics Against Different Cancers

According to a story from finanznachrichten.de, the biotherapeutics company 4D pharma has recently announced a research partnership with the MD Anderson Cancer Center at the University of Texas. The goal of…

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