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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Teenage Girl Walks Again After Finally Getting Diagnosed With Complement C1q Deficiency and Bone Marrow Transplant
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Teenage Girl Walks Again After Finally Getting Diagnosed With Complement C1q Deficiency and Bone Marrow Transplant

  A young girl in Mauritius, an island in the Indian Ocean, has recently been given a second chance at a normal life after receiving a rare disease diagnosis and…

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Gene Therapy Proves Effective in Treating AADC Deficiency in Trial
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Gene Therapy Proves Effective in Treating AADC Deficiency in Trial

According to the publication over the weekend from San Francisco CBS affiliate KPIX, a groundbreaking clinical trial for those with aromatic l-amino acid decadboxylase (AADC) deficiency was undertaken at UCSF…

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New Cholesterol-Lowering Drug Could Provide Alternative to Statins
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New Cholesterol-Lowering Drug Could Provide Alternative to Statins

According to a publication from EurekAlert, high-cholesterol patients, who do not take statins (a type of cholesterol-lowering medication often prescribed by doctors) due to adverse side effects may benefit from…

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People Impacted by Hansen’s Disease in Latin America Unite After Historic Conference
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People Impacted by Hansen’s Disease in Latin America Unite After Historic Conference

According to a story from the Inter Press Service, the First Latin American and Caribbean Assembly of Organisations of People Affected by Hansen's Disease has just recently concluded. Hansen's disease,…

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Man With Urea Cycle Disorder is the First Person in Canada to be Treated With Intravenous Gene Therapy
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Man With Urea Cycle Disorder is the First Person in Canada to be Treated With Intravenous Gene Therapy

According to a story from ctvnews.ca, a recent treatment with gene therapy appears to have changed the life of thirty year old Josh McQuillin, who has a urea cycle disorder,…

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