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    Rett Syndrome - 2 year(s) ago

    Rett syndrome is a multisystem disorder that primarily affects girls; multiple loss-of-function mutations to the MECP2 gene are the cause of this rare disease.

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    • Check out our learning page on Rett Syndrome @Rettsyndrome @rettsyndnews #checkrare #rarediseases #rettsyndrome https://t.co/0xP5CrvwIY

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    The LAVENDER Trial - 2 year(s) ago

    Dr. Ponni Subbiah discusses the results of the LAVENDER trial, which led to approval of trofinetide (DAYBUE) in patients with Rett syndrome.

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    • Ponni Subbiah, MD, MPH, Chief Medical Officer at Acadia Pharmaceuticals, discusses the results of the LAVENDER trial, which led to the approval of trofinetide #DAYBUE in patients with Rett syndrome #checkrare #rettsyndrome @rettsyndnews @Rettsyndrome https://t.co/oSABIjwcyv

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    Believe in Brynn - 2 year(s) ago

    Mr. Shawn Connor, father of a girl with Rett syndrome, talks about their advocacy group, Believe in Brynn.

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    • Shawn Connor, father of a girl with Rett syndrome, talks about the advocacy group, Believe in Brynn #checkrare #rarediseases #rettsyndrome @believeinbrynn @rettsyndnews https://t.co/Jt9cl2Jt6y

  • Mashup Score: 0
    Rett Syndrome - 2 year(s) ago

    Rett syndrome is a multisystem disorder that primarily affects girls; multiple loss-of-function mutations to the MECP2 gene are the cause of this rare disease.

    Tweet Tweets with this article
    • Rett syndrome is a multisystem disorder that primarily affects girls. Only in rare cases are boys affected (who may experience more severe symptoms). Multiple loss-of-function mutations to the MECP2 gene are the cause of #Rettsyndrome @rettsyndnews https://t.co/0xP5Crw4yw

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    Laura Mamounas PhD, Program Director at the National Institute of Neurological Disorder and Stroke (NINDS) at the NIH in Bethesda, discusses Rett syndrome research at her organization. Rett syndrome is a multisystem disorder that primarily affects girls. Only in rare cases are boys affected (who may experience more severe symptoms). Multiple loss-of-function mutations to […]

    Tweet Tweets with this article
    • Laura Mamounas, Ph.D., Program Director at the National Institute of Neurological Disorder and Stroke #NINDS at the NIH in Bethesda, discusses Rett syndrome research at her organization #checkrare #rarediseases #rettsyndrome @NIH @ncats_nih_gov https://t.co/f03nMsLUrK

  • Mashup Score: 0
    Believe in Brynn - 2 year(s) ago

    Mr. Shawn Connor, father of a girl with Rett syndrome, talks about their advocacy group, Believe in Brynn.

    Tweet Tweets with this article
    • Shawn Connor, father of a girl with Rett syndrome, talks about the advocacy group, Believe in Brynn #checkrare #rarediseases #rettsyndrome @believeinbrynn @rettsyndnews https://t.co/Jt9cl2Jt6y