News

November 17, 2020November 17, 2020

Bryostatin-1 in Long-term Use Seen to Arrest Fragile X Symptoms in Mouse Model

News

Long-term, but not short-term, treatment with bryostatin-1 — Neurotrope’s lead investigational therapy — arrested such behavioral and cognitive symptoms as hyperactivity, difficulties with daily life activities, and learning and ... Read more

November 13, 2020November 13, 2020

Study Seeking to Link Brain Processes With Cognition, Behavior Now Recruiting

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A Phase 1 clinical trial is recruiting adults with fragile X syndrome to study the brain processes underlying altered behavior and cognition, according to the FRAXA Research Foundation. Conducted ... Read more

November 6, 2020November 6, 2020

Fragile X Protein May Be Crucial for Cells That Support Neurons, Study Finds

News

Loss of the fragile X mental retardation protein (FMRP), the protein missing in people with fragile X syndrome, was found in mouse and human astroglia — nerve cells that support ... Read more

November 5, 2020November 6, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit ... Read more

November 3, 2020November 3, 2020

BPN14770 Shows Cognition Benefits in Phase 2 Clinical Trial

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The investigational treatment BPN14770 improved language skills, cognition, and caregiver-assessed daily functioning ability in adults with fragile X syndrome, top-line results of a Phase 2 clinical trial show. “We ... Read more

October 30, 2020October 30, 2020

Screening for Fragile X Carriers May Be Cost-free Option for Couples in Australia

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The Australian Medical Services Advisory Committee (MSAC) has recommended that the costs of screening for mutations known to cause cystic fibrosis (CF), spinal muscular atrophy (SMA), and fragile X ... Read more

October 29, 2020October 29, 2020

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

News, syndicated

A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar ... Read more

October 27, 2020October 27, 2020

FMRP Protein Helps Prevent DNA Damage, Maintain Genome Stability, Study Finds

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The fragile X mental retardation protein (FMRP) — the protein missing in people with fragile X syndrome — helps prevent DNA damage and maintain genome stability, a study found. According ... Read more

October 23, 2020October 23, 2020

Phase 2 Trial Planned for Xanamem, Oral Treatment for Fragile X

News

Actinogen Medical announced plans to open a Phase 2 trial of its experimental oral therapy Xanamem in boys with fragile X syndrome early next year. The study, to be called XanaFX ... Read more

October 23, 2020October 23, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

News, syndicated

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a ... Read more