New Clinical Trial Application for Alpha-1 Antitrypsin Deficiency-Associated Liver Disease Submitted in Sweden

Dicerna Pharmaceuticals has just announced that they have submitted a Clinical Trial Authorization application for their investigational therapy DCR-A1AT. This application has been submitted to the Swedish Medical Products Agency…

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Company Starts up Late Stage Trial Testing Experimental WHIM Syndrome Treatment
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Company Starts up Late Stage Trial Testing Experimental WHIM Syndrome Treatment

According to a story from BioPortfolio, the biopharmaceutical company X4 Pharmaceuticals, Inc. has recently initiated a phase 3 clinical trial that is testing its current lead product candidate mavorixafor. This…

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Researchers Observe First Cases of Congenital Leptin Deficiency in the Americas
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Researchers Observe First Cases of Congenital Leptin Deficiency in the Americas

According to a press release from the Texas Biomedical Research Institute (Texas Biomed), researchers from the Institute have identified another new genetic mutation that they believe can cause rare congenital…

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Canadian Study Indicates That Prescribing Practices of Infliximab for Ulcerative Colitis Need Amendment

Inflammatory Bowel Diseases Inflammatory bowel diseases (IBD) is the overarching term for both ulcerative colitis and Crohn's disease. In Canada, there are around 270,000 individuals living with the one of…

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CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients
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CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients

According to a press release from the California Institute for Regenerative Medicine (CIRM), the Institute's governing body has approved a grant of nearly $12 million to University of California, San…

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The IND Approval From The FDA, Experimental Acromegaly Drug Will Enter Phase 3 Trials
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The IND Approval From The FDA, Experimental Acromegaly Drug Will Enter Phase 3 Trials

According to a story from Markets Insider, the biopharmaceutical company Camurus has recently issued an announcement indicating that the US Food and Drug Administration (FDA) has accepted the company's Investigational…

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An Addison’s Disease Story: Multiple Rare Diagnoses Often Means a Longer Waiting Game

Emily Dover  This is Emily Dover's story. At birth- perfectly healthy newborn, smaller than her siblings (8 pounds) 1 week- was having trouble sleeping and began crying in pain 4…

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Study Finds Poor Adherence to PDE-5I Drugs Can Have Consequences in Pulmonary Arterial Hypertension
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Study Finds Poor Adherence to PDE-5I Drugs Can Have Consequences in Pulmonary Arterial Hypertension

According to a story from Pulmonary Hypertension News, a recent study has found that lapses in treatment adherence can result in serious side effects for pulmonary arterial hypertension (PAH) patients…

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How do You Find Strength as Amyotrophic Lateral Sclerosis Causes Your Body to Weaken?

According to a story from princetoninfo.com, Sara Cooper was diagnosed last November with amyotrophic lateral sclerosis. Sara has had an active career as an event planner, marketing consultant, and real…

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Chronic Lymphocytic Leukemia: Patterns of Treatment, Costs, and Adverse Events
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Chronic Lymphocytic Leukemia: Patterns of Treatment, Costs, and Adverse Events

According to a story from Cancer Therapy Advisor, a recent study published in the journal Cancer Medicine indicated that chronic lymphocytic leukemia (CLL) patients who dealt with adverse events during treatment…

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