Fragile X News Today

October 23, 2020October 23, 2020

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

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 and ... Read more

October 20, 2020October 20, 2020

Zygel May Improve Behavior in Children With Severe Fragile X, Trial Data Suggest

Fragile X News Today

Zygel, an experimental cannabidiol (CBD) gel, may lessen the extent of behavioral abnormalities in children with fragile X syndrome who have more severe disease, new clinical trial data suggest. The ... Read more

October 16, 2020October 16, 2020

UC Davis Establishing 4-Year Program for Students With Fragile X, Other Disabilities

Fragile X News Today

The U.S. Department of Education awarded $2.1 million to the University of California, Davis, MIND Institute to create a four-year college program for students with intellectual disabilities, including those with ... Read more

October 13, 2020October 13, 2020

Patrick Wild Centre Opens Website for Its Fragile X Registry

Fragile X News Today

The Patrick Wild Centre at The University of Edinburgh launched an online portal for its fragile X syndrome registry on Oct. 10, European fragile X awareness day. The new website, called eHub, ... Read more

October 9, 2020October 9, 2020

Connecta Therapeutics Raises Funds to Advance Potential Fragile X Therapy

Fragile X News Today

Connecta Therapeutics raised €1.7 million (about $2 million) in investments that will enable it to bring a potential fragile X syndrome (FXS) therapy to Phase 1 clinical trials in late ... Read more

October 6, 2020October 6, 2020

$25M NIH Grant to Support Fragile X Research at 3 Centers

Fragile X News Today

The National Institutes of Health (NIH) is awarding multi-year grants to three centers conducting collaborative research into fragile X syndrome: Baylor College of Medicine, Emory University, and Cincinnati Children’s Hospital ... Read more

September 25, 2020September 25, 2020

Fragile X Girls Less Aware of Bullying, Study Suggests

Fragile X News Today

School-aged girls with fragile X syndrome are less aware of bullying than children of the same age and intellectual abilities, a study suggests. These findings indicate that difficulties in interpreting ... Read more

September 22, 2020September 22, 2020

Strong Placebo Effect Warrants Better Trial Design in Fragile X, Study Finds

Fragile X News Today

Clinical improvements among participants receiving a placebo — known as the placebo effect — are common in clinical trials of fragile X syndrome, a study conducted by scientists across the ... Read more

September 18, 2020September 18, 2020

Hospitalization After Emergency Department Visit Common in Fragile X Patients, Study Finds

Fragile X News Today

Almost half of emergency department visits for fragile X syndrome result in hospitalizations, according to a nationwide study in the U.S. The finding underscores the importance of surveillance systems capable ... Read more

September 15, 2020September 15, 2020

Zynerba, FDA to Discuss Next Steps for Zygel in Fragile X Treatment

Fragile X News Today

The U.S. Food and Drug Administration (FDA) and Zynerba Pharmaceuticals will meet later this year to discuss the results of the CONNECT-FX trial and next steps regarding a potential regulatory ... Read more

September 11, 2020September 11, 2020

Closing Leaky Mitochondria Halts Behavioral Problems in Fragile X

Fragile X News Today

Shortage of energy due to leaky mitochondria — the powerhouses of cells — contributes to behavioral abnormalities in fragile X syndrome, a study has found. Closure of the leaky mitochondrial ... Read more

September 8, 2020October 9, 2020

Newly Discovered Mechanisms Controlling Memory, Learning Could Lead to Therapies

Fragile X News Today

Scientists have discovered part of the mechanisms underlying synaptic plasticity — the process that allows nerve cell connections called synapses to become stronger or weaker over time — that are ... Read more

August 28, 2020September 29, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Fragile X News Today

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input ... Read more

August 25, 2020August 26, 2020

Better Communication Needed to Help Fragile X Patients Transition Into Adulthood

Fragile X News Today

Better communication among different institutions is needed to assist young patients with fragile X syndrome make the transition from school into adult life, according to a small study. Support programs ... Read more

August 21, 2020August 21, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Fragile X News Today

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The ... Read more