Author Archives: Marisa Wexler MS

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Beyond Batten Disease Foundation (BBDF) has struck a deal with Actelion Pharmaceuticals to help further the development of BBDF-101, an investigational treatment for juvenile Batten disease. Under the terms of the new agreement, Actelion (a Janssen Pharmaceutical Company of Johnson & Johnson) will provide a component of…

New guidelines from the U.S. Food and Drug Administration (FDA) may make it easier to develop therapies to treat extremely rare diseases, such as Batten disease. “The release of this guidance from the FDA is a first step and a very welcome one. It speaks to the promise…

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 organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

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 series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

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 — has been described in the first published case of these two conditions occurring in one person. The case report,…

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 property developed by researchers at the University of North Carolina at Chapel Hill (UNC) and Abeona,…

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 — which seeks to slow the progression of the inherited neurological condition — pursuant to…

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 gene therapy for Batten disease to receive orphan drug designation from the FDA this summer.

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 serves as a measure of CLN3 disease severity,” was published in JIMD Reports. Vacuoles are compartments…