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Disorders and Conditions

New Gene Therapy for "Bubble Boy" Disease Appears to be Safe, Effective

PHILADELPHIA — A new form of gene therapy for boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a life-threatening condition also known ...

HEALTH LINE: Thrombosis Shouldn't Be Taken Lightly

CINCINNATI—Venous thromboembolism isn't as well known as stroke or heart attack, but this cardiovascular foe can be just as deadly. ...

Researchers discover potential early-detection biomarker for 'bubble boy' disorder

BLACKSBURG, Va. – Many people recognize “the bubble boy” as an unusual character from a “Seinfeld” episode or a John Travolta movie. ... Full story

New Zealand ahead in self-managing common conditions

New Zealand and the United Kingdom are the most progressive countries for empowering consumers to self-manage common conditions, according to recent research. ... Full story

Cardiorespiratory fitness is often misdiagnosed

A recent study by the University of Eastern Finland shows that scaling maximal oxygen uptake and maximal workload by body weight confounds measures of cardiorespiratory fitness. ... Full story

Behçet’s syndrome drugs to be investigated for first time

The University of Liverpool is to lead and international study to investigate the effectiveness of two drugs used to treat a rare but serious disease, in a project that will save the NHS a minimum of £500k. ... Full story

FDA approves U-M invented drug for Gaucher disease

ANN ARBOR—Cerdelga, a treatment for Gaucher disease type 1 licensed by the University of Michigan to Genzyme Corp., has been approved by the Food and Drug Administration. ... Full story

First patient trials new treatment for rare inherited disease

Researchers at The University of Manchester and The Royal Manchester Children’s Hospital have recruited the first child into a new study, which aims to evaluate the clinical effectiveness of a treatment developed in Manchester. ... Full story

New information on transcranial ultrasound therapy

A recent study completed at the University of Eastern Finland provides new information on the limitations and potential new directions for the future development of transcranial ultrasound therapy. ... Full story

New study to improve leg ulcer treatment

Sport and exercise scientists at Sheffield Hallam University have started a new study into the treatment of Chronic Venous Insufficiency (CVI), which affects patients' legs, often causing painful leg ulcers. ... Full story

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