Phase 3 Myelodysplastic Syndromes Trial Reaches Enrollment Milestone
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Phase 3 Myelodysplastic Syndromes Trial Reaches Enrollment Milestone

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company's Phase 3…

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Internet-Based Therapy Could Aid Chronic Fatigue Syndrome Patients
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Internet-Based Therapy Could Aid Chronic Fatigue Syndrome Patients

According to a study published by the Journal of Medical Internet research (JMIR), patients diagnosed with chronic fatigue syndrome (CFS) who are treated with cognitive behavioral therapy (CBT) delivered over…

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Results from Three Clinical Trials for Urea Cycle Disorders and Cirrhosis Coming by the End of the Year
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Results from Three Clinical Trials for Urea Cycle Disorders and Cirrhosis Coming by the End of the Year

Three non-investigational New Drug clinical studies are underway for Urea Cycle Disorders (UCD) and various forms of cirrhosis, initiated by Kaleido Biosciences. Dosing has been initiated in each of these…

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Extremely Positive Results from Systemic Juvenile Idiopathic Arthritis 5-year Follow-Up Study in the EU

Extremely positive results have just been announced from a 5-year follow-up study of a systemic juvenile idiopathic arthritis (sJIA) treatment called Kineret. This treatment has been approved for use in…

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Two Companies Collaborate on Progressive Supranuclear Palsy Clinical Trial
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Two Companies Collaborate on Progressive Supranuclear Palsy Clinical Trial

According to a story from BioPortfolio, the biopharmaceutical company AlzProtect recently announced that PAREXEL Biotech, which is a subsidiary of PAREXEL International Corporation, will be expected to conduct a Phase…

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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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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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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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