News

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 two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Compared to children whose vision loss is due to Stargardt disease, those with  juvenile Batten (CLN3) disease have more extensive retinal pathology leading to a distinctively more rapid loss of visual acuity, color blindness, and poor retinal responses to light, a…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

Taysha Gene Therapies announced that it has joined with Catalent, a pharmaceutical services provider, to support production of its potential gene therapies for disorders that include CLN1 Batten disease and Rett syndrome. “Through this partnership, we will be able to enhance our existing manufacturing capabilities to support Taysha’s broad…

Adding cannabis oil to standard anti-seizure medications may be an effective way of managing treatment-resistant status epilepticus — life-threatening seizures that require urgent medical treatment — in people with CLN6 Batten disease, a case report suggests. This…

Neurogene has enrolled the first patient in a natural history study of CLN5 and CLN7 disease, two forms of late-infantile Batten disease, the company announced. The study (NCT03822650), which is still recruiting, is being led by Jonathan Mink, MD, PhD, director of the University of Rochester Batten Disease…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

The U.S. Food and Drug Administration (FDA) has given a favorable opinion of the continued preclinical development plan for BBDF-101, a potential new treatment for juvenile Batten disease (CLN3). The plan, made by Theranexus and the Beyond Batten Disease Foundation (BBDF), supports efforts to submit an…