A Treatment for Thrombotic Microangiopathy Has Been Awarded Breakthrough Status by the F.D.A.
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A Treatment for Thrombotic Microangiopathy Has Been Awarded Breakthrough Status by the F.D.A.

The US Food and Drug Administration (FDA) has just awarded Breakthrough Therapy Designation to a drug (OMS721) being developed to treat thrombotic microangiopathy that has developed as a result of haematopoietic stem…

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A Gene Therapy Being Developed to Treat Batten Disease Has Been Granted Orphan Drug Status in Europe
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A Gene Therapy Being Developed to Treat Batten Disease Has Been Granted Orphan Drug Status in Europe

A gene therapy called ABO-202 that is being developed to treat Batten disease has just been granted Orphan Drug Designation by the European Medicines Agency (EMA), reports BioPortfolio. This follows…

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Research into a Genetic Mutation Has Led to the Discovery of a Rare Disease
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Research into a Genetic Mutation Has Led to the Discovery of a Rare Disease

A group of researchers led by Professor Gholson Lyon has discovered a genetic mutation that is linked to certain traits, reports Medical Xpress. The method used to discover it was…

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A Treatment Trial Has Produced Promising Results in Patients With Myelodysplastic Syndromes
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A Treatment Trial Has Produced Promising Results in Patients With Myelodysplastic Syndromes

A press release from Aprea Theraputics has just announced the results of a Phase II clinical trial of the drug APR-246 in patients with myelodysplastic syndromes (a group of blood…

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A New QR-Code System Will Improve Emergency Care for Patients With Addison’s Disease
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A New QR-Code System Will Improve Emergency Care for Patients With Addison’s Disease

Researchers from Newcastle University in the UK have just developed a potentially life-saving device for patients with Addison’s disease, reports Newcastle University. Patients will be given a Quick Response (QR)…

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Anavex Plans to Trial its Treatment For Parkinson’s Disease Dementia, and Researchers are Hopeful that it Could Also Benefit Other Diseases
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Anavex Plans to Trial its Treatment For Parkinson’s Disease Dementia, and Researchers are Hopeful that it Could Also Benefit Other Diseases

A drug called Anavex 2-73 being developed to treat Parkinson’s disease dementia is planned to undergo testing in a Phase 2 clinical trial, reports Globe Newswire. Parkinson’s disease is a…

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A Common Virus is a Risk Factor for Seven Serious Diseases
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A Common Virus is a Risk Factor for Seven Serious Diseases

A study carried out by researchers at Cincinnati Children’s Hospital has discovered that the extremely common Epstein-Barr virus is a risk factor for seven diseases, including lupus and multiple sclerosis.…

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A New Study Has Identified Why Patients With Hemophilia Have Such High Rates of Prosthetic Joint Infection and What Can be Done About It
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A New Study Has Identified Why Patients With Hemophilia Have Such High Rates of Prosthetic Joint Infection and What Can be Done About It

  The Orthopaedic Institute for Children (OIC) has found that improving the education of patients with hemophilia leads to a large reduction in rates of prosthetic joint infection, reports Bioportfolio.…

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Study Suggests That Kidney Disease Researchers Aren’t Considering Patient-Focused Outcomes
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Study Suggests That Kidney Disease Researchers Aren’t Considering Patient-Focused Outcomes

According to a story from PR Newswire, a recent study indicated that researchers that were focused on kidney illnesses such as chronic kidney disease, congenital nephrotic syndrome, and glomerulonephritis have not been…

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