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    Rare Diseases

    Rare Diseases: A disease or disorder is defined as rare in Europe when it affects fewer than 1 in 2000. A disease or disorder is defined as rare in the USA when it affects fewer than 200,000 Americans at any given time. ... In the EU, as many as 30 million people may be affected by one of over 6000 existing rare diseases.

    Trending News

    • JUN 20, 2019
      Drug Discovery & Development
      Sickle cell drug promising in clinical trial
      Sickle cell drug promising in clinical trial
      UConn School of Medicine researchers report that an investigational drug used for treating for the sickle cell disease h ...
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      2
      JUN 18, 2019
      Drug Discovery & Development
      Drug Compound May Treat Common Genetic Kidney Disease
      Drug Compound May Treat Common Genetic Kidney Disease
      Researchers from the University of Sheffield in collaboration with Glasgow-based biotech company Mironid, have developed ...
      Written By: Nouran Amin
      2
      JUN 17, 2019
      Microbiology
      Patient Dies After Fecal Transplant
      Patient Dies After Fecal Transplant
      Fecal transplants are still considered an experimental procedure, a fact highlighted by two recent infections.
      Written By: Carmen Leitch
      1
      JUN 11, 2019
      Technology
      Artificial Intelligence Can Help Detect Rare Diseases
      Artificial Intelligence Can Help Detect Rare Diseases
      An estimated half a million children worldwide are born with a rare hereditary disease. To obtain a definitive diagnosis ...
      Written By: Nouran Amin
      1
      JUN 10, 2019
      Cell & Molecular Biology
      Finding a Way to Predict Epileptic Seizures
      Finding a Way to Predict Epileptic Seizures
      Right now, there's no way to know when epileptic seizures will occur, and they could happen at dangerous times.
      Written By: Carmen Leitch
      1
      JUN 10, 2019
      Drug Discovery & Development
      Novel Treatments for Auto-immune Disorders
      Novel Treatments for Auto-immune Disorders
      A recent research study examined a library of almost 300,000 small molecules to search for a molecule that may be a pote ...
      Written By: Nouran Amin
      1
      JUN 05, 2019
      Microbiology
      Surgery Reveals That Supposed Brain Tumor is a Parasite
      Surgery Reveals That Supposed Brain Tumor is a Parasite
      A woman began having terrible nightmares, hallucinations, and insomnia. Doctors did an MRI and thought it was a brain tu ...
      Written By: Carmen Leitch
      1
      MAY 29, 2019
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      The Link Between Uterine Fibroids And Cardiovascular Disease
      The Link Between Uterine Fibroids And Cardiovascular Disease
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      MAY 29, 2019
      Health & Medicine
      Novel Gene Therapy Approved by FDA to Treat Spinal Muscular Atrophy in Pediatric Patients
      Novel Gene Therapy Approved by FDA to Treat Spinal Muscular Atrophy in Pediatric Patients
      The most common form of spinal muscular atrophy (SMA), a rare genetic disease, is caused by mutations of chromosome ...
      Written By: Dena Aruta
      1
      MAY 23, 2019
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      Miracle Drug for Rare Genetic Disease Causes Price Concerns
      Miracle Drug for Rare Genetic Disease Causes Price Concerns
      A newly approved “miracle drug” called Zolgensma will be used to treat children with spinal muscular atrophy ...
      Written By: Nouran Amin
      1
      MAY 23, 2019
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      The Genetic Answer to Bland Tomatoes
      The Genetic Answer to Bland Tomatoes
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      MAY 13, 2019
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      Bud Rot: When Marijuana Gets Moldy
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      MAY 07, 2019
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      Siblings lead Researchers to Discover New Immunodeficiency Disease
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      MAR 28, 2019
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      Big Pharma to Take Another Look at Marijuana
      Big Pharma to Take Another Look at Marijuana
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      MAR 27, 2019
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      A Role for the Endocannabinoid System in Down Syndrome
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      MAR 17, 2019
      Genetics & Genomics
      Increasing Evidence Shows Narcolepsy is an Autoimmune Illness
      Increasing Evidence Shows Narcolepsy is an Autoimmune Illness
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      MAR 06, 2019
      Health & Medicine
      A Second Patient Cured of HIV: Are We Close to a Cure for Everyone Infected?
      A Second Patient Cured of HIV: Are We Close to a Cure for Everyone Infected?
      There is a consensus that the first HIV infection occurred in the 1920s in Kinshasa, the Democratic Republic o ...
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      MAR 04, 2019
      Health & Medicine
      European Respiratory Society Task Force Sarcoidosis Patient Survey
      European Respiratory Society Task Force Sarcoidosis Patient Survey
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      3
      FEB 27, 2019
      Immunology
      When the Cell Dials 9-1-1
      When the Cell Dials 9-1-1
      Researchers can now explain how a cell that is being attacked by bacteria or viruses specifically manages to 'sound the ...
      Written By: Nicholas Breehl
      2
      FEB 25, 2019
      Drug Discovery & Development
      3D Hydrogel Advances Drug Discovery
      3D Hydrogel Advances Drug Discovery
      Researchers have developed a 3D hydrogel based on hyaluronic acid, a biocompatible substance. The gel could quickly and ...
      Written By: Nouran Amin
      1
      FEB 25, 2019
      Drug Discovery & Development
      Open-Science Model Advances Drug Discovery
      Open-Science Model Advances Drug Discovery
      Parkinson’s disease and Amyotrophic Lateral Sclerosis is the latest push for Medicines 4 Neurodegenerative Disease ...
      Written By: Nouran Amin
      1
      FEB 19, 2019
      Cell & Molecular Biology
      Slowing the Aging Process
      Slowing the Aging Process
      It's inevitable in life, but aging isn't really something people look forward to.
      Written By: Carmen Leitch
      1
      FEB 12, 2019
      Immunology
      Why Does My Throat Hurt Again
      Why Does My Throat Hurt Again
      Scientists are uncovering the mechanisms behind recurring cases of step throat
      Written By: Nicholas Breehl
      2
      FEB 12, 2019
      Cancer
      Humans and Man's Best Friend Share Genetic Mutations in Rare Form of Melanoma
      Humans and Man's Best Friend Share Genetic Mutations in Rare Form of Melanoma
      A recent study compared the cancer genomes of a rare form of melanoma (mucosal melanoma) in humans, dogs, and horses in ...
      Written By: Amy Loriaux
      1
    JUN 20, 2019
    Drug Discovery & Development
    Sickle cell drug promising in clinical trial
    Sickle cell drug promising in clinical trial
    UConn School of Medicine researchers report that an investigational drug used for treating for the sickle cell disease h ...
    Written By: Nouran Amin
    2
    JUN 18, 2019
    Drug Discovery & Development
    Drug Compound May Treat Common Genetic Kidney Disease
    Drug Compound May Treat Common Genetic Kidney Disease
    Researchers from the University of Sheffield in collaboration with Glasgow-based biotech company Mironid, have developed ...
    Written By: Nouran Amin
    2
    JUN 17, 2019
    Microbiology
    Patient Dies After Fecal Transplant
    Patient Dies After Fecal Transplant
    Fecal transplants are still considered an experimental procedure, a fact highlighted by two recent infections.
    Written By: Carmen Leitch
    1
    JUN 11, 2019
    Technology
    Artificial Intelligence Can Help Detect Rare Diseases
    Artificial Intelligence Can Help Detect Rare Diseases
    An estimated half a million children worldwide are born with a rare hereditary disease. To obtain a definitive diagnosis ...
    Written By: Nouran Amin
    1
    JUN 10, 2019
    Cell & Molecular Biology
    Finding a Way to Predict Epileptic Seizures
    Finding a Way to Predict Epileptic Seizures
    Right now, there's no way to know when epileptic seizures will occur, and they could happen at dangerous times.
    Written By: Carmen Leitch
    1
     
     
  • JUN 10, 2019
    Drug Discovery & Development
    Novel Treatments for Auto-immune Disorders
    Novel Treatments for Auto-immune Disorders
    A recent research study examined a library of almost 300,000 small molecules to search for a molecule that may be a pote ...
    Written By: Nouran Amin
    1
    JUN 05, 2019
    Microbiology
    Surgery Reveals That Supposed Brain Tumor is a Parasite
    Surgery Reveals That Supposed Brain Tumor is a Parasite
    A woman began having terrible nightmares, hallucinations, and insomnia. Doctors did an MRI and thought it was a brain tu ...
    Written By: Carmen Leitch
    1
    MAY 29, 2019
    Cardiology
    The Link Between Uterine Fibroids And Cardiovascular Disease
    The Link Between Uterine Fibroids And Cardiovascular Disease
    Uterine fibroids are generally benign tumors of the smooth muscle within the uterus. In females, they are the most commo ...
    Written By: Abbie Arce
    1
    MAY 29, 2019
    Health & Medicine
    Novel Gene Therapy Approved by FDA to Treat Spinal Muscular Atrophy in Pediatric Patients
    Novel Gene Therapy Approved by FDA to Treat Spinal Muscular Atrophy in Pediatric Patients
    The most common form of spinal muscular atrophy (SMA), a rare genetic disease, is caused by mutations of chromosome ...
    Written By: Dena Aruta
    1
    MAY 23, 2019
    Drug Discovery & Development
    Miracle Drug for Rare Genetic Disease Causes Price Concerns
    Miracle Drug for Rare Genetic Disease Causes Price Concerns
    A newly approved “miracle drug” called Zolgensma will be used to treat children with spinal muscular atrophy ...
    Written By: Nouran Amin
    1
     
     
  • MAY 23, 2019
    Genetics & Genomics
    The Genetic Answer to Bland Tomatoes
    The Genetic Answer to Bland Tomatoes
    Every year, the world consumes around 182 million tons of tomatoes. Yet, thanks to years of genetic editing and crossbre ...
    Written By: Annie Lennon
    1
    MAY 13, 2019
    Cannabis Sciences
    Bud Rot: When Marijuana Gets Moldy
    Bud Rot: When Marijuana Gets Moldy
    With increases in marijuana production, a particular problem has begun to emerge: mold. Cannabis is actually very suscep ...
    Written By: Amy Loriaux
    2
    MAY 07, 2019
    Health & Medicine
    Siblings lead Researchers to Discover New Immunodeficiency Disease
    Siblings lead Researchers to Discover New Immunodeficiency Disease
    An infant was suffering from inflammatory bowel disease, eczema, food allergies, lung disease, and persistent cytomegalo ...
    Written By: Tiffany Dazet
    2
    MAR 28, 2019
    Cannabis Sciences
    Big Pharma to Take Another Look at Marijuana
    Big Pharma to Take Another Look at Marijuana
    Since the FDA approved of the first drug derived from cannabis last year, pharmaceutical companies big and small are tak ...
    Written By: Amy Loriaux
    1
    MAR 27, 2019
    Cannabis Sciences
    A Role for the Endocannabinoid System in Down Syndrome
    A Role for the Endocannabinoid System in Down Syndrome
    Scientists out of Spain led by Dr. Andrés Ozaita revealed last month in their publication in the journal&nbs ...
    Written By: Amy Loriaux
    2
     
     
  • MAR 17, 2019
    Genetics & Genomics
    Increasing Evidence Shows Narcolepsy is an Autoimmune Illness
    Increasing Evidence Shows Narcolepsy is an Autoimmune Illness
    Narcolepsy is a chronic sleep disorder impacting as many as 200,000 people in the US, and is thought to often go undiagn ...
    Written By: Carmen Leitch
    2
    MAR 06, 2019
    Health & Medicine
    A Second Patient Cured of HIV: Are We Close to a Cure for Everyone Infected?
    A Second Patient Cured of HIV: Are We Close to a Cure for Everyone Infected?
    There is a consensus that the first HIV infection occurred in the 1920s in Kinshasa, the Democratic Republic o ...
    Written By: Dena Aruta
    2
    MAR 04, 2019
    Health & Medicine
    European Respiratory Society Task Force Sarcoidosis Patient Survey
    European Respiratory Society Task Force Sarcoidosis Patient Survey
    Sarcoidosis is a rare, granulomatous disease that is found in the lungs of 90% of people diagnosed, but it can affect an ...
    Written By: Dena Aruta
    3
    FEB 27, 2019
    Immunology
    When the Cell Dials 9-1-1
    When the Cell Dials 9-1-1
    Researchers can now explain how a cell that is being attacked by bacteria or viruses specifically manages to 'sound the ...
    Written By: Nicholas Breehl
    2
    FEB 25, 2019
    Drug Discovery & Development
    3D Hydrogel Advances Drug Discovery
    3D Hydrogel Advances Drug Discovery
    Researchers have developed a 3D hydrogel based on hyaluronic acid, a biocompatible substance. The gel could quickly and ...
    Written By: Nouran Amin
    1
     
     
  • FEB 25, 2019
    Drug Discovery & Development
    Open-Science Model Advances Drug Discovery
    Open-Science Model Advances Drug Discovery
    Parkinson’s disease and Amyotrophic Lateral Sclerosis is the latest push for Medicines 4 Neurodegenerative Disease ...
    Written By: Nouran Amin
    1
    FEB 19, 2019
    Cell & Molecular Biology
    Slowing the Aging Process
    Slowing the Aging Process
    It's inevitable in life, but aging isn't really something people look forward to.
    Written By: Carmen Leitch
    1
    FEB 12, 2019
    Immunology
    Why Does My Throat Hurt Again
    Why Does My Throat Hurt Again
    Scientists are uncovering the mechanisms behind recurring cases of step throat
    Written By: Nicholas Breehl
    2
    FEB 12, 2019
    Cancer
    Humans and Man's Best Friend Share Genetic Mutations in Rare Form of Melanoma
    Humans and Man's Best Friend Share Genetic Mutations in Rare Form of Melanoma
    A recent study compared the cancer genomes of a rare form of melanoma (mucosal melanoma) in humans, dogs, and horses in ...
    Written By: Amy Loriaux
    1
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