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June 5, 2020June 5, 2020

Specific Piece of FMRP Protein Eases Hyperactivity in Mouse Model

News

Providing a fragment of the FMRP protein, which is missing in people with fragile X syndrome, eased hyperactivity and restored nerve cell communication in the brain, a study in ... Read more

June 2, 2020June 2, 2020

Shionogi Acquires Tetra Therapeutics, Rights to Fragile X Treatment Candidate

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Shionogi has acquired Tetra Therapeutics, the developer of a treatment candidate known as BPN14770 for fragile X syndrome and Alzheimer’s disease. With the agreement, Shionogi obtains global rights to Tetra’s entire pipeline. It will ... Read more

May 28, 2020May 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

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While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene ... Read more

May 26, 2020May 26, 2020

Blocking GSK Alpha Enzyme Safely Reverses Fragile X Symptoms in Mouse Model

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Blocking an enzyme called GSK3 alpha seems to ease many symptoms of fragile X syndrome — including abnormal protein production, seizures induced by loud tones, learning and memory deficits, and ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

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European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session ... Read more

May 21, 2020May 22, 2020

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

cystic fibrosis, News, syndicated

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s ... Read more

May 20, 2020May 20, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

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Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. ... Read more

May 19, 2020May 19, 2020

Trial Results for Zygel, CBD Gel Treatment for Fragile X, Likely by Mid-year

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Top-line results from the CONNECT-FX trial testing the cannabidiol (CBD) gel Zygel as a potential treatment for behavioral symptoms in children and teenagers with fragile X syndrome are expected by late ... Read more

May 12, 2020May 12, 2020

OV101 Well-tolerated, Effective in Fragile X Patients, Phase 2 Trial Shows

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Treatment with OV101 (gaboxadol) was well-tolerated and significantly eased behavioral and functional symptoms in people with fragile X syndrome, top-line data from a Phase 2 clinical trial show. The results support ... Read more

May 11, 2020May 11, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

News, syndicated

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. ... Read more

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