“I’m 38. I Can’t Have Parkinson’s:” A Chronic Illness Story From the Czech Republic
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“I’m 38. I Can’t Have Parkinson’s:” A Chronic Illness Story From the Czech Republic

It started with fatigue. Then came problems with moving, hand tremors and problems walking. Multiple sclerosis was ruled out by magnetic resonance imaging (MRI). But the trouble didn’t go away.…

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Study Suggests That Heritable Parkinson’s Disease is More Prevalent Than Expected

According to a story from Parkinson's News Today, the results of a global, online survey appear to indicate that genetically based, inherited Parkinson's disease is actually more common than the…

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Enrollment Begins for Amyotrophic Lateral Sclerosis Clinical Trial
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Enrollment Begins for Amyotrophic Lateral Sclerosis Clinical Trial

According to a story from BioPortfolio, the biopharmaceutical company Collaborative Medicinal Development, LLC has recently announced that the first patient has been enrolled in a clinical trial that will test…

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FDA Approves Supplementary Treatment For Parkinson’s Disease “Off” Episodes
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FDA Approves Supplementary Treatment For Parkinson’s Disease “Off” Episodes

A recent publication from the US Food and Drug Administration announced the regulatory authority's approval of Nourianz (generic name istradefylline) for Parkinson's patients during "off" episodes. "Off" episodes are windows…

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Researchers Might Have Just Made a Breakthrough Discovery for Treating Rare Proteinopathies

It's common knowledge among researchers who study diseases called toxic proteinopathies that this group of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…

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Researchers are Working to Repair a Small Step In a Pathway Involved in Neurodegenerative Diseases

It's common knowledge among researchers who study diseases called toxic proteinopathies, that this grou of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…

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This Time a “First-of-its-Kind” Study is “Out of this World” on Board the Space Station

  As reported recently in PhysOrg by NASA, researchers aboard SpaceX CRS-18 cargo flight are focusing on what triggers Parkinson’s disease and multiple sclerosis, both neurodegenerative diseases. Researchers suspect that…

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A Brief Discussion of How Cell and Gene Therapies are Being Improved for Future Generations

  While the last few years have seen major advances in all areas of the pharmaceutical industry, some of the newest and brightest talents are now focusing on cell and…

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DNA Sequencing Took Years, Now it Takes Hours; As a Result, Researchers Have Discovered The Cause of Four Rare Diseases

Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…

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King’s College Research Suggests Serotonin Levels Could Predict Parkinson’s Years Before Symptoms
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King’s College Research Suggests Serotonin Levels Could Predict Parkinson’s Years Before Symptoms

According to a publication from the BBC, researchers with King's College London believe they've identified early biomarkers for Parkinson's disease that could help physicians predict who will be affected up…

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A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body
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A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body

  The blood-brain barrier is a “gatekeeper” that prevents toxins and foreign matter in the bloodstream from traveling to the brain. Check Orphan recently carried an article that had been…

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The FDA is Reversing Course on Unapproved Products Sold by Stem Cell Manufacturers

  The FDA’s “untitled letter” to R3 Stem Cell (R3), based in Scottsdale Arizona, cautioned R3 that the product they are marketing is considered a drug. It must, therefore, be…

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The FDA, Congress, A Young Woman Dying of ALS, Her Physician, and Her Parents Are All Struggling Over Access to an Untested Therapy

  The Hermstads of Spencer, Iowa lost Jaci’s twin to ALS and now they must watch Jaci, rapidly descend into the depths of the same disease. Jaci has a rare…

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The PoppyPocket Helps Patients Wear Their Infusion Pumps More Comfortably, Safely, and Confidently

The PoppyPocket The PoppyPocket is a wearable pocket, created to make wearing infusion pumps and well as other medical devices easier, safer, and more comfortable. It comes with two pockets,…

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ALZHEIMER’S:  Scientists are Back to Square One; Will Bryostatin-1 be the Missing Link?

  Pharmaceutical companies have spent billions of dollars on Alzheimer’s research. The target has been the toxic protein, amyloid-beta. According to a recent article in NPR News, this theory was…

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