Posts by: Marisa Wexler, MS

Marisa Wexler, MS

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

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 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

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 who have more severe disease, new clinical trial data suggest. ... Read more

October 6, 2020October 6, 2020

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

News

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 ... Read more

September 22, 2020September 22, 2020

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

News

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 ... Read more

July 24, 2020July 24, 2020

Zygel Improves Caregiver Impressions of Behavioral Symptoms in Some Fragile X Children

News

The experimental cannabidiol (CBD) gel Zygel eased behavioral problems in children with fragile X syndrome and benefited those with greater disease severity according to their caregivers, new clinical trial data show. The ... Read more

July 17, 2020July 17, 2020

Metformin Seen to Help Correct Defects in Neurons Lacking FMRP Protein

News

The common type 2 diabetes medication metformin eased the excessive production of proteins in nerve cells that were engineered in a lab to have the underlying defect seen in people with ... Read more

July 7, 2020July 7, 2020

Zygel May Be Effective for Children with Severe Fragile X Symptoms, CONNECT-FX Trial Suggests

News

Zynerba Pharmaceuticals‘ Zygel failed to significantly ease behavioral symptoms in the overall group of children with fragile X syndrome (FXS) who took part in the CONNECT-FX trial, top-line results ... Read more

May 19, 2020May 19, 2020

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

News

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