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June 23, 2020June 23, 2020

Parent Language Intervention Eases Speech, Social Impairments in Fragile X, Trial Finds

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A parent language intervention based on story-telling eases the severity of speech and social impairments in children with fragile X syndrome, a study shows. The study also found that ... Read more

June 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

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The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will ... Read more

June 19, 2020June 19, 2020

Inflammation Not a Hallmark of Fragile X, Study Suggests

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Low levels of pro-inflammatory proteins in the blood suggest that inflammation is not a hallmark of fragile X syndrome, according to a new study. The findings also indicated that assessing the ... Read more

June 12, 2020June 12, 2020

Trofinetide Safe and Effective at Higher Dose for Fragile X, Phase 2 Trial Finds

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Oral treatment with trofinetide was well tolerated and, at a higher dose, eased social avoidance, anxiety, hyperactivity and other symptoms of fragile X syndrome in adolescent and adult males, ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

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The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis ... Read more

June 9, 2020June 9, 2020

Genetic Screening Study Starts Recruiting Prospective Parents in Australia

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An Australian project is recruiting couples planning to have children or experiencing early pregnancy for a nationwide genetic study to screen for about 750 genetic conditions, including fragile X ... Read more

June 5, 2020June 5, 2020

Specific Piece of FMRP Protein Eases Hyperactivity in Mouse Model

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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

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