November 20, 2020November 20, 2020
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry
News, syndicated
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how ... Read more
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 ... Read more
November 13, 2020November 13, 2020
Study Seeking to Link Brain Processes With Cognition, Behavior Now Recruiting
News
A Phase 1 clinical trial is recruiting adults with fragile X syndrome to study the brain processes underlying altered ... 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 ... 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 ... Read more
November 3, 2020November 3, 2020
BPN14770 Shows Cognition Benefits in Phase 2 Clinical Trial
News
The investigational treatment BPN14770 improved language skills, cognition, and caregiver-assessed daily functioning ability in adults with fragile X syndrome, ... Read more
October 30, 2020October 30, 2020
Screening for Fragile X Carriers May Be Cost-free Option for Couples in Australia
News
The Australian Medical Services Advisory Committee (MSAC) has recommended that the costs of screening for mutations known to cause ... 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 ... Read more
October 27, 2020October 27, 2020
FMRP Protein Helps Prevent DNA Damage, Maintain Genome Stability, Study Finds
News
The fragile X mental retardation protein (FMRP) — the protein missing in people with fragile X syndrome — helps prevent ... 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 ... 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 ... Read more
October 20, 2020October 20, 2020
Zygel May Improve Behavior in Children With Severe Fragile X, Trial Data Suggest
News
Zygel, an experimental cannabidiol (CBD) gel, may lessen the extent of behavioral abnormalities in children with fragile X syndrome ... Read more

November 20, 2020November 20, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about ... Read more

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

News

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

News

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

News

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

News

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