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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

    • NOV 20, 2018
      Genetics & Genomics
      Scientists Link Three Genes to Miscarriage
      Scientists Link Three Genes to Miscarriage
      A miscarriage can be a devastating experience, and researchers are trying to learn more about genetic factors that may i ...
      Written By: Carmen Leitch
      1
      NOV 19, 2018
      Microbiology
      The Gut Microbiome Changes Dramatically When Hunter-gatherers Start Farming
      The Gut Microbiome Changes Dramatically When Hunter-gatherers Start Farming
      Researchers have shown that lifestyle changes have a rapid and significant influence on the human gut microbiome.
      Written By: Carmen Leitch
      2
      NOV 17, 2018
      Genetics & Genomics
      Joubert Syndrome Model Successfully Treated with Gene-editing
      Joubert Syndrome Model Successfully Treated with Gene-editing
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      NOV 15, 2018
      Cannabis Sciences
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      Marijuana-Induced Cyclic Vomiting
      Trigger warning: If you are a bit squeamish about emesis (vomiting) then this article may not be for you. Otherwise ...
      Written By: Amy Loriaux
      1
      NOV 13, 2018
      Drug Discovery & Development
      Clinical Trial Drug For a Rare Neurodegenrative Disease Proven Unsuccessful
      Clinical Trial Drug For a Rare Neurodegenrative Disease Proven Unsuccessful
      Niemann-Pick type C (NPC) is a rare and fatal neurodegenerative disease. Symptoms of the condition include loss of balan ...
      Written By: Nouran Amin
      2
      NOV 12, 2018
      Cancer
      Targeted radiation therapy may provide new hope to children with difficult-to-treat liver cancer
      Targeted radiation therapy may provide new hope to children with difficult-to-treat liver cancer
      Primary malignant liver tumors are rare in children with an occurrence rate of 1-2% of all childhood cancers. Radical su ...
      Written By: Heba El-wassef
      1
      NOV 07, 2018
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      The Threat of Neglected Tropical Diseases
      The Threat of Neglected Tropical Diseases
      Did you know that around one billion people suffer from a neglected tropical disease?
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      NOV 06, 2018
      Genetics & Genomics
      New Gene is Implicated in a Rare Cancer
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      NOV 05, 2018
      Microbiology
      Potential Antidote to Botulism is Found
      Potential Antidote to Botulism is Found
      A microbe called Clostridium botulinum and sometimes two other strains of Clostridium bacteria can make a toxic chemical ...
      Written By: Carmen Leitch
      1 2
      NOV 01, 2018
      Cell & Molecular Biology
      Researchers Link Parkinson's Disease and the Appendix
      Researchers Link Parkinson's Disease and the Appendix
      When a person's appendix is removed early in life, it reduces their chances of getting Parkinson's disease.
      Written By: Carmen Leitch
      1
      OCT 31, 2018
      Drug Discovery & Development
      New Drug Target For a Rare Lymphoma
      New Drug Target For a Rare Lymphoma
      The promise of immunotherapy in oncology is stronger than ever. Now, an international team of scientists and physicians ...
      Written By: Nouran Amin
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      OCT 23, 2018
      Immunology
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      Missense Immune Disruption
      A point mutation has caused a de novo immune deficiency observed in two patients
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      OCT 21, 2018
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      The Genetic Cause of Severe Childhood Epilepsy is Revealed
      The Genetic Cause of Severe Childhood Epilepsy is Revealed
      This work has opened up new therapeutic avenues that the scientists will be exploring.
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      1
      OCT 18, 2018
      Genetics & Genomics
      Expanding the List of Genes That Cause Multiple Sclerosis
      Expanding the List of Genes That Cause Multiple Sclerosis
      For many years, researchers have been searching for the genetic influences that affect the development of MS.
      Written By: Carmen Leitch
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      OCT 08, 2018
      Microbiology
      Is a Virus Causing a Mysterious Polio-like Illness in Kids?
      Is a Virus Causing a Mysterious Polio-like Illness in Kids?
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      OCT 01, 2018
      Neuroscience
      What is Lewy Body Dementia?
      What is Lewy Body Dementia?
      Dementia is a scary diagnosis, but what many people don't realize is that dementia is a general term that can refer ...
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      1
      SEP 29, 2018
      Microbiology
      In a First, Rat Variation of Hepatitis E Found in a Person
      In a First, Rat Variation of Hepatitis E Found in a Person
      It was found in a 56-year-old Hong Kong man.
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      SEP 08, 2018
      Drug Discovery & Development
      Protein Associated With ALS Inspires Drug Treatments
      Protein Associated With ALS Inspires Drug Treatments
      Neurodegenerative diseases, such as degenerative ataxia or amyotrophic lateral sclerosis (ALS), will progressively cause ...
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      AUG 31, 2018
      Videos
      One Class of Diabetes Drugs Can Increase Risk of Genital Gangrene
      One Class of Diabetes Drugs Can Increase Risk of Genital Gangrene
      The FDA has issued a serious warning about a group of type 2 diabetes drug called sodium-glucose cotransporter-2 (SGLT2) ...
      Written By: Carmen Leitch
      1
      AUG 31, 2018
      Immunology
      B Cells Control Connective Tissue Disorder Scleroderma
      B Cells Control Connective Tissue Disorder Scleroderma
      Effector and Regulatory B cells play reciprocal role in pathogenesis of scleroderma.
      Written By: Caitlin Williams
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      AUG 27, 2018
      Cardiology
      Drug Reduces Deaths and Hospitalization from Heart Failure
      Drug Reduces Deaths and Hospitalization from Heart Failure
      New drug reduces deaths and hospitalization rates, while also improving quality of life, for patients with rare type of ...
      Written By: Caitlin Williams
      1
      AUG 26, 2018
      Genetics & Genomics
      Why the Same Genetic Mutation can Affect People Differently
      Why the Same Genetic Mutation can Affect People Differently
      Scientists have been confounded for years by genetic errors with the sequence, but a different effect on people.
      Written By: Carmen Leitch
      1
      AUG 15, 2018
      Immunology
      Therapeutic Macrophages Improve Rare Lung Disorder
      Therapeutic Macrophages Improve Rare Lung Disorder
      The use of therapeutic macrophage transplantation in mice showed great improvement in rare lung disorder caused by defic ...
      Written By: Caitlin Williams
      1
      AUG 05, 2018
      Genetics & Genomics
      Newly ID'ed Lung Cell Changes Our Understanding of Disease
      Newly ID'ed Lung Cell Changes Our Understanding of Disease
      This rare type of cell seems to play a major role in cystic fibrosis.
      Written By: Carmen Leitch
      1
    NOV 20, 2018
    Genetics & Genomics
    Scientists Link Three Genes to Miscarriage
    Scientists Link Three Genes to Miscarriage
    A miscarriage can be a devastating experience, and researchers are trying to learn more about genetic factors that may i ...
    Written By: Carmen Leitch
    1
    NOV 19, 2018
    Microbiology
    The Gut Microbiome Changes Dramatically When Hunter-gatherers Start Farming
    The Gut Microbiome Changes Dramatically When Hunter-gatherers Start Farming
    Researchers have shown that lifestyle changes have a rapid and significant influence on the human gut microbiome.
    Written By: Carmen Leitch
    2
    NOV 17, 2018
    Genetics & Genomics
    Joubert Syndrome Model Successfully Treated with Gene-editing
    Joubert Syndrome Model Successfully Treated with Gene-editing
    A life-threatening kidney disease may be one day be a treatable condition thanks to new work by researchers, and patient ...
    Written By: Carmen Leitch
    2
    NOV 15, 2018
    Cannabis Sciences
    Marijuana-Induced Cyclic Vomiting
    Marijuana-Induced Cyclic Vomiting
    Trigger warning: If you are a bit squeamish about emesis (vomiting) then this article may not be for you. Otherwise ...
    Written By: Amy Loriaux
    1
    NOV 13, 2018
    Drug Discovery & Development
    Clinical Trial Drug For a Rare Neurodegenrative Disease Proven Unsuccessful
    Clinical Trial Drug For a Rare Neurodegenrative Disease Proven Unsuccessful
    Niemann-Pick type C (NPC) is a rare and fatal neurodegenerative disease. Symptoms of the condition include loss of balan ...
    Written By: Nouran Amin
    2
     
     
  • NOV 12, 2018
    Cancer
    Targeted radiation therapy may provide new hope to children with difficult-to-treat liver cancer
    Targeted radiation therapy may provide new hope to children with difficult-to-treat liver cancer
    Primary malignant liver tumors are rare in children with an occurrence rate of 1-2% of all childhood cancers. Radical su ...
    Written By: Heba El-wassef
    1
    NOV 07, 2018
    Videos
    The Threat of Neglected Tropical Diseases
    The Threat of Neglected Tropical Diseases
    Did you know that around one billion people suffer from a neglected tropical disease?
    Written By: Carmen Leitch
    1
    NOV 06, 2018
    Genetics & Genomics
    New Gene is Implicated in a Rare Cancer
    New Gene is Implicated in a Rare Cancer
    Rodents are known as common research models, but zebrafish have been gaining ground as an attractive alternative for man ...
    Written By: Carmen Leitch
    1 1
    NOV 05, 2018
    Microbiology
    Potential Antidote to Botulism is Found
    Potential Antidote to Botulism is Found
    A microbe called Clostridium botulinum and sometimes two other strains of Clostridium bacteria can make a toxic chemical ...
    Written By: Carmen Leitch
    1 2
    NOV 01, 2018
    Cell & Molecular Biology
    Researchers Link Parkinson's Disease and the Appendix
    Researchers Link Parkinson's Disease and the Appendix
    When a person's appendix is removed early in life, it reduces their chances of getting Parkinson's disease.
    Written By: Carmen Leitch
    1
     
     
  • OCT 31, 2018
    Drug Discovery & Development
    New Drug Target For a Rare Lymphoma
    New Drug Target For a Rare Lymphoma
    The promise of immunotherapy in oncology is stronger than ever. Now, an international team of scientists and physicians ...
    Written By: Nouran Amin
    3
    OCT 23, 2018
    Immunology
    Missense Immune Disruption
    Missense Immune Disruption
    A point mutation has caused a de novo immune deficiency observed in two patients
    Written By: Nicholas Breehl
    1
    OCT 21, 2018
    Genetics & Genomics
    The Genetic Cause of Severe Childhood Epilepsy is Revealed
    The Genetic Cause of Severe Childhood Epilepsy is Revealed
    This work has opened up new therapeutic avenues that the scientists will be exploring.
    Written By: Carmen Leitch
    1
    OCT 18, 2018
    Genetics & Genomics
    Expanding the List of Genes That Cause Multiple Sclerosis
    Expanding the List of Genes That Cause Multiple Sclerosis
    For many years, researchers have been searching for the genetic influences that affect the development of MS.
    Written By: Carmen Leitch
    1
    OCT 08, 2018
    Microbiology
    Is a Virus Causing a Mysterious Polio-like Illness in Kids?
    Is a Virus Causing a Mysterious Polio-like Illness in Kids?
    Sometime in 2014, the CDC began to get reports of a mysterious illness that was affecting children. More cases have appe ...
    Written By: Carmen Leitch
    1
     
     
  • OCT 01, 2018
    Neuroscience
    What is Lewy Body Dementia?
    What is Lewy Body Dementia?
    Dementia is a scary diagnosis, but what many people don't realize is that dementia is a general term that can refer ...
    Written By: Brenda Kelley Kim
    1
    SEP 29, 2018
    Microbiology
    In a First, Rat Variation of Hepatitis E Found in a Person
    In a First, Rat Variation of Hepatitis E Found in a Person
    It was found in a 56-year-old Hong Kong man.
    Written By: Carmen Leitch
    1
    SEP 08, 2018
    Drug Discovery & Development
    Protein Associated With ALS Inspires Drug Treatments
    Protein Associated With ALS Inspires Drug Treatments
    Neurodegenerative diseases, such as degenerative ataxia or amyotrophic lateral sclerosis (ALS), will progressively cause ...
    Written By: Nouran Amin
    1
    AUG 31, 2018
    Videos
    One Class of Diabetes Drugs Can Increase Risk of Genital Gangrene
    One Class of Diabetes Drugs Can Increase Risk of Genital Gangrene
    The FDA has issued a serious warning about a group of type 2 diabetes drug called sodium-glucose cotransporter-2 (SGLT2) ...
    Written By: Carmen Leitch
    1
    AUG 31, 2018
    Immunology
    B Cells Control Connective Tissue Disorder Scleroderma
    B Cells Control Connective Tissue Disorder Scleroderma
    Effector and Regulatory B cells play reciprocal role in pathogenesis of scleroderma.
    Written By: Caitlin Williams
    1
     
     
  • AUG 27, 2018
    Cardiology
    Drug Reduces Deaths and Hospitalization from Heart Failure
    Drug Reduces Deaths and Hospitalization from Heart Failure
    New drug reduces deaths and hospitalization rates, while also improving quality of life, for patients with rare type of ...
    Written By: Caitlin Williams
    1
    AUG 26, 2018
    Genetics & Genomics
    Why the Same Genetic Mutation can Affect People Differently
    Why the Same Genetic Mutation can Affect People Differently
    Scientists have been confounded for years by genetic errors with the sequence, but a different effect on people.
    Written By: Carmen Leitch
    1
    AUG 15, 2018
    Immunology
    Therapeutic Macrophages Improve Rare Lung Disorder
    Therapeutic Macrophages Improve Rare Lung Disorder
    The use of therapeutic macrophage transplantation in mice showed great improvement in rare lung disorder caused by defic ...
    Written By: Caitlin Williams
    1
    AUG 05, 2018
    Genetics & Genomics
    Newly ID'ed Lung Cell Changes Our Understanding of Disease
    Newly ID'ed Lung Cell Changes Our Understanding of Disease
    This rare type of cell seems to play a major role in cystic fibrosis.
    Written By: Carmen Leitch
    1
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