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 6, 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

October 20, 2020October 20, 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 9, 2020October 9, 2020

First Report Published of Batten and Moyamoya Diseases Occurring in Same Patient

News

A patient with both Batten disease and Moyamoya disease — a condition where blood vessels in the skull become obstructed or narrowed, restricting blood flow to the brain — ... Read more

September 11, 2020September 11, 2020

Abeona Partners With Taysha to Advance ABO-202 as Potential Gene Therapy

News

Abeona Therapeutics and Taysha Gene Therapies have entered into agreements concerning ABO-202, an investigative gene therapy for infantile Batten disease. The agreements give Taysha worldwide exclusive rights to intellectual ... Read more

August 28, 2020August 28, 2020

FDA Names Investigational Therapy BBDF-101 Orphan Drug, Rare Pediatric Disease

News

The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation and rare pediatric disease designation to BBDF-101, an investigational treatment for Batten disease. Theranexus is developing BBDF-101 ... Read more

August 21, 2020August 21, 2020

Neurogene’s CLN7 Gene Therapy Granted FDA Orphan Drug Designation

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a gene therapy being developed by Neurogene for the treatment of CLN7 Batten disease. This marks the second Neurogene ... Read more

July 17, 2020July 17, 2020

Lymphocyte Vacuolization Might Be Used to Measure Batten Severity

News

The number of fluid-containing cellular compartments called vacuoles within immune cells could help to predict the severity of Batten disease, a new study suggests. The study, “Quantifying lymphocyte vacuolization ... Read more

June 5, 2020June 5, 2020

Pakistani Study Describes New Mutations That Cause CLN5 Disease

News

Two previously-unknown mutations in the CLN5 gene were reported to cause CLN5 disease, a form of late infantile Batten disease, in a new study. The study also represents the ... Read more

May 15, 2020May 15, 2020

Amicus’ CLN3 Batten Disease Gene Therapy Gets FDA Fast Track Designation

News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Amicus Therapeutics‘ AT-GTX-502, an investigational gene therapy for Batten disease caused by mutations in the CLN3 ... Read more

April 10, 2020April 10, 2020

FDA Favors Clinical Trial of Potential Juvenile Batten Treatment, PLX-200

News

Polaryx Therapeutics has received approval from the U.S. Food and Drug Administration (FDA) to open a clinical trial into PLX-200 as a potential treatment for juvenile Batten disease. Juvenile Batten, ... Read more