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C4 Genes as a Double-Edged Sword: Sex-Based Differences in Lupus, Schizophrenia, and Sjögren’s Syndrome

C4 Genes as a Double-Edged Sword: Sex-Based Differences in Lupus, Schizophrenia, and Sjögren’s Syndrome

  Women can do anything that men can do, and vice versa. Well, while that sentiment may be true in our everyday life, it isn't quite true on a genetic…

Continue Reading C4 Genes as a Double-Edged Sword: Sex-Based Differences in Lupus, Schizophrenia, and Sjögren’s Syndrome
Review of Rare Movement Disorders in India and the United States

Review of Rare Movement Disorders in India and the United States

A recent article published in India’s Pharma Literati cites The World Health Organization as not having endorsed a definition for rare diseases. The European Union defines 'rare' as less than…

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Gene Therapy: The New Frontier for Cancer

Gene Therapy: The New Frontier for Cancer

  Gene therapy is the treatment of disease by way of the transfer of genetic material into cells. According to a recent article in Labiotech, an EU publication, gene therapy…

Continue Reading Gene Therapy: The New Frontier for Cancer
‘Bosentan’ Approved by FDA to Treat Pulmonary Arterial Hypertension

‘Bosentan’ Approved by FDA to Treat Pulmonary Arterial Hypertension

As originally reported in Global Data, the FDA has just given the final approval for a treatment options for pulmonary arterial hypertension (PAH) called 'Bosentan.' Pulmonary Arterial Hypertension Pulmonary Arterial Hypertension…

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ICYMI: New Form of Gene Editing Could Correct 89% of Genetic Defects

ICYMI: New Form of Gene Editing Could Correct 89% of Genetic Defects

We've been hearing A LOT about so-called 'gene editing' and 'CRISPR' lately -- and now there's new developments on that front. Scientists have developed a new gene-editing technology called 'prime…

Continue Reading ICYMI: New Form of Gene Editing Could Correct 89% of Genetic Defects
New Prime Editing Has The Potential to Search and Replace up to 89% of Genetic Defects

New Prime Editing Has The Potential to Search and Replace up to 89% of Genetic Defects

  CNN recently covered a story in the publication Nature about a paper explaining base editing (or prime editing). The researchers who created the technology set forth the process of using base…

Continue Reading New Prime Editing Has The Potential to Search and Replace up to 89% of Genetic Defects
Editor’s Choice: Equality, Advocacy, and a Sense of Purpose in the Rare Community

Editor’s Choice: Equality, Advocacy, and a Sense of Purpose in the Rare Community

Happy Thursday! Today, we're highlighting stories on three patients facing different struggles: a professor, a Jeopardy host, and a man from New Jersey. We also have an article about a…

Continue Reading Editor’s Choice: Equality, Advocacy, and a Sense of Purpose in the Rare Community
Eight Children with Severe Combined Immunodeficiency Cured with Gene Therapy

Eight Children with Severe Combined Immunodeficiency Cured with Gene Therapy

Severe Combined Immunodeficiency Severe Combined Immunodeficiency (SCID) is a rare disease which causes patients to practically have no immune system at all. That means that for these patients, even a…

Continue Reading Eight Children with Severe Combined Immunodeficiency Cured with Gene Therapy
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Finding Light Through Story-The Power of Ambassadorship in the Endometrial Cancer Community
*cancer that cannot be removed with surgery or has spread to other parts of the body. GEJ= gastroesophageal junction (where the esophagus joins the stomach)
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