Forest Ray, PhD,  —

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…

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…

Cannabinoids were used to successfully treat seizures in a patient with Batten disease for whom conventional therapies alone failed, according to a case report, suggesting that cannabinoids should be investigated further as a possible seizure treatment. The report, “Successful treatment of cannabinoid administration against refractory…

The European Medicines Agency (EMA) has given priority medicines (PRIME) status to AT-GTX-501, Amicus Therapeutics‘ investigational gene therapy for CLN6 Batten disease, otherwise known as variant late infantile neuronal ceroid lipofuscinosis 6 (vLINCL6) disease. PRIME designation helps speed the development and regulatory review of promising medicines…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

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 into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.

Those afflicted with lysosomal storage disorders such as Batten disease may benefit from a clever new treatment strategy one day. A recent study has demonstrated how to turn a certain antibody into a so-called Trojan horse for ferrying healthy enzymes missing in these disorders across the blood-brain barrier. This…