Prophylaxis May Reduce the Need of Hospitalization for von Willebrand Disease Patients, Study Says
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Prophylaxis May Reduce the Need of Hospitalization for von Willebrand Disease Patients, Study Says

According to a story from the National Hemophilia Foundation, a collaborative team of researchers from the U.S. and Sweden recently announced the results of a study the indicated that a…

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Clinical Trials Are in the Works That Will Test a Treatment for Phelan-McDermid Syndrome
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Clinical Trials Are in the Works That Will Test a Treatment for Phelan-McDermid Syndrome

According to a story from Newswise, the Seaver Autism Center for Research and Treatment at Mount Sinai recently announced that patient enrollment has begun for a clinical trial that will…

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What are Nano Ghosts? This Woman May Have Uncovered One of the Greatest Breakthroughs in Cancer Treatment
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What are Nano Ghosts? This Woman May Have Uncovered One of the Greatest Breakthroughs in Cancer Treatment

According to a story from The Jerusalem Post, the medical research of professor Marcelle Machluf has been a subject of controversy from the beginning. Her research led to the nano…

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By Observing the Effects of Alagille Syndrome, Researchers Discover How Liver Tissue Can Transform and Heal
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By Observing the Effects of Alagille Syndrome, Researchers Discover How Liver Tissue Can Transform and Heal

According to a story from PR Newswire, a group of scientists have managed to discover a mechanism that allows the liver to repair damage it has sustained. This discovery may…

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Researchers Design Compact Infection Detector That Can be Used With a Smart Phone
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Researchers Design Compact Infection Detector That Can be Used With a Smart Phone

According to a story from bioengineer.org, scientists from Washington State University have successfully developed a compact and cost effective infection detection device that is comparable in effectiveness to clinical labs.…

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Anti-Inflammatory Effects of Baking Soda Could Help Fight Rheumatoid Arthritis and Chronic Kidney Disease
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Anti-Inflammatory Effects of Baking Soda Could Help Fight Rheumatoid Arthritis and Chronic Kidney Disease

According to a story from Healthline, a daily regimen of baking soda consumption could help reduce the severity of inflammation in certain diseases, such as chronic kidney disease and rheumatoid…

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After a Dangerous Accident and CIDP, a Girl Shows her Courage
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After a Dangerous Accident and CIDP, a Girl Shows her Courage

According to a story from the Chillicothe Gazette, twelve-year-old Brooke Hopkins has already had to face chronic inflammatory demyelinating polyneuropathy and a devastating injury in her young life. However, through…

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Study Testing Self-Administration of Hereditary Angioedema Treatment Shows Positive Results
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Study Testing Self-Administration of Hereditary Angioedema Treatment Shows Positive Results

According to a story from Rare Disease Report, a recent study indicates that the self-administration of Berinert, a drug used to treat hereditary angioedema, is effective and safe for patients…

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The University of Southern California’s Keck School of Medicine Just Got Funding to Research Rare Lung Diseases
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The University of Southern California’s Keck School of Medicine Just Got Funding to Research Rare Lung Diseases

According to a story from HSC News, the Hastings Foundation recently gave a gift of $12.5 million for the USC Hastings Center for Pulmonary Research, a subdivision of the University…

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Investigational Treatment for hATTR Amyloidosis Shows Promising Results in Trials
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Investigational Treatment for hATTR Amyloidosis Shows Promising Results in Trials

According to a story from Rare Disease Report, the pharmaceutical company Alnylam Pharmaceuticals announced the results of Phase 3 trials for its experimental drug patisiran. The drug is in the…

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Most Epilepsy Patients Benefit From Current Medications, But Look Forward to New Treatments

According to a story from Scimex, a recent assessment of available treatments for Dravet syndrome and other types of epilepsy has reaffirmed that anticonvulsant medications are still the most effective…

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ICYMI: Experimental Drug for Spinal Muscular Atrophy Gets Orphan Drug Designation
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ICYMI: Experimental Drug for Spinal Muscular Atrophy Gets Orphan Drug Designation

According to a story from BioSpace, the pharmaceutical company Scholar Rock recently announced that its drug candidate SRK-015 was recently granted Orphan Drug Designation by the U.S. FDA. The drug…

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Communicating and Connecting With Others Can Improve Quality of Life in FCS Patients
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Communicating and Connecting With Others Can Improve Quality of Life in FCS Patients

According to a story from the Rare Disease Report, the results of a recent survey questionnaire indicate that communicating with other rare disease patients can cause a significant improvement in…

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