Treatment for Childhood and Spinal Cord Cancer Linked to Lower Levels of Independence for Survivors
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Treatment for Childhood and Spinal Cord Cancer Linked to Lower Levels of Independence for Survivors

  The survival rates for infants and children with brain and spinal cord cancer has improved significantly over the past decades. However, a study published online in early August of…

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Aplastic Anemia and MDS International Foundation: 2018 Scientific Symposium
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Aplastic Anemia and MDS International Foundation: 2018 Scientific Symposium

At the 2018 Aplastic Anemia and MDS International Foundation’s (AAMDSIF) International Bone Marrow Failure Disease Scientific Symposium of March 2018, Dr. David Margolis from the Medical College of Wisconsin, and…

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New Statement Released by the FH Foundation on Genetic Testing for Familial Hypercholesterolemia

According to a story from the FH Foundation, the non-profit organization has released a vital consensus statement regarding the diagnosis of familial hypercholesterolemia, which is the most prevalent genetic cause…

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A Man With Spinal Muscular Atrophy is Stuck Without Access to Costly Treatment
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A Man With Spinal Muscular Atrophy is Stuck Without Access to Costly Treatment

According to a story from cbc.ca, Cole Pringle is a 31-year-old man from Regina, Saskatchewan with spinal muscular atrophy, a debilitating illness that severely affects his mobility. He was diagnosed…

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Tauopathy Fruit Fly Model Reveals Abnormal Genetic Activity Relevant to Neurodegenerative Disease
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Tauopathy Fruit Fly Model Reveals Abnormal Genetic Activity Relevant to Neurodegenerative Disease

According to a public release from University of Texas Health Center at San Antonio on eurekalert.org, research using fruit fly tauopathy disease models has revealed a genetic "copy-paste" activity that…

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Could This Mouth Swab Save Lives? Genepath Test Diagnosis Krabbe Disease and Much More
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Could This Mouth Swab Save Lives? Genepath Test Diagnosis Krabbe Disease and Much More

Ashley Wibberly’s son Levi was born in September of 2013 and lived for just 20 months with Krabbe disease. Ashley couldn’t have seen it coming. Now, however, a new test…

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October 2018 is the Expected Date for Top-Line Results From a Phase 3 Trial of an Experimental Treatment for Charcot-Marie-Tooth Disease
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October 2018 is the Expected Date for Top-Line Results From a Phase 3 Trial of an Experimental Treatment for Charcot-Marie-Tooth Disease

An ongoing Phase 3 clinical trial program is investigating the drug PXT3003 as an experimental treatment for Charcot-Marie-Tooth disease type 1A in adults. Pharnext SA says that they expect to…

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