Researchers Ask Hemophilia Patients to Evaluate Their Quality of Life on Study Questionnaire
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Researchers Ask Hemophilia Patients to Evaluate Their Quality of Life on Study Questionnaire

According to a publication from Hemophilia News Today, an international team of researchers with members from France and the United Kingdom recently conducted a study of over 100 hemophilia patients…

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Researchers Find Gut Bacteria that Can Change Type A Blood to Type O
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Researchers Find Gut Bacteria that Can Change Type A Blood to Type O

Blood Donations For many people living with a rare disease such as Beta thalassemia, Hemophilia, Aplastic Anemia, Myelodysplastic syndromes, Acute Promyelocytic leukemia, and others, blood transfusions are not uncommon. For…

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Experimental Hemophilia A Drug Loses Effectiveness Over Time, Efficacy Questionable After 8th Year

According to a publication from Reuters, based on early clinical data, American biotechnology company BioMarin Pharmaceuticals believes that a single injection of its experimental hemophilia A drug could relieve symptoms…

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Loma Linda University Cures Their First Sickle Cell Disease Patient with Stem Cell Transplant

LLUCH Loma Linda University Children's Hospital (LLUCH) is one of six hospitals in the University's health system. They are a faith-based system located in Southern California. They are the only…

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uniQure Receives FDA Fast Track Designation for AMT 130 Gene Therapy for the Treatment of Huntingtons Disease

LEXINGTON, Mass. and AMSTERDAM, the Netherlands - uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs,  announced that the U.S. Food and Drug…

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The Struggles of being a Rare Disease Patient in China

The State of Rare Diseases in China The Illness Challenge Foundation (ICF) is an organization devoted to easing the burdens that rare disease patients living in China face. They accomplish this…

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Preventive Use of Extended Half-Life Therapies Provides Enhanced Benefit for Hemophilia A Patients
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Preventive Use of Extended Half-Life Therapies Provides Enhanced Benefit for Hemophilia A Patients

According to a story from BioSpace, a recent study revealed that hemophilia A patients were less likely to experience bleeds when they used new, extended half-life therapies on a preventive…

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Secondary Long-Term Prophylaxis Shows Promise for Patients with Severe von Willebrand Disease (vWD)

A recent article in the National Hemophilia Foundation news quotes the Blood Transfusion journal's report on the results of a study conducted under the lead of Dr. Flora Peyvandi, Medical…

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Loneliness in a Rare Disease Diagnosis: A Different Kind of Co-Morbidity

Rare diseases such as Tourette's syndrome and cystic fibrosis can be, among all of the other challenges that come with their diagnosis, extremely isolating. A recent article on Psychology Today explains…

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Updates From Spark Highlight the Potential of Gene Therapy for Treating Hemophilia

According to a story from Hemophilia News Today, the drug developer Spark Therapeutics, Inc., has recently released several updates in regards to a number of its experimental gene therapies that…

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Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects
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Passage Bio Receives New Funding to Advance 5 Gene Therapy Projects

Passsage Bio Passage Bio has just announced that they've received 115.5 million dollars to help advance their development of gene therapies. They will begin by using the funds to further…

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Press Release: uniQure Announces First Patient Treated in HOPE-B Pivotal Trial of AMT-061 in Patients with Hemophilia B

Full Patient Enrollment in Study Expected by Year-End 2019 LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 04, 2019 -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for…

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Doctor Sells Teenager Plasma as a “Miracle Cure” but Lacks Proof
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Doctor Sells Teenager Plasma as a “Miracle Cure” but Lacks Proof

According to an article originally published by HuffPost, an ambitious 34-year-old doctor is peddling plasma transfusions at steep prices, claiming a number of dubious health benefits. Ambrosia, LLC In 2014…

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The Latest in St. Jude Research on Blood Cancers and Blood Disorders
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The Latest in St. Jude Research on Blood Cancers and Blood Disorders

According to a story from EurekAlert!, the 60th Annual Meeting of the American Society of Hematology featured a number of different presentations of the latest research from St. Jude Children's…

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