A new mutation in the KCTD7 gene was identified in a boy as the cause of CLN14 disease, a rare form…
Articles by Patricia Inácio, PhD
The University of Rochester Batten Center (URBC) was designated a Center of Excellence by the Batten Disease Support, Research, and…
A first patient has been dosed in a Phase 1/2 clinical trial testing the gene therapy RGX-381 — one…
Delivering a CLN5-targeted gene therapy simultaneously to the brain and eye in a sheep model of late infantile Batten disease…
Use of the cholesterol medication gemfibrozil — a fat-lowering therapy — reduced brain inflammation and improved motor function in…
Treatment for up to two months with Batten-1 — Theranexus’ novel therapy for juvenile Batten disease, also known…
Theranexus and the Beyond Batten Disease Foundation (BBDF) have completed enrollment for a Phase 1/2 clinical trial testing Batten-1…
Amicus Therapeutics announced it will discontinue its investigational gene therapy program of AT-GTX-501 for late infantile neuronal ceroid lipofuscinosis 6 disease, also…
Rarebase, a public benefit biotechnology company, has launched the Function therapeutic discovery platform specifically for rare neurological diseases, including…
The Beyond Batten Disease Foundation (BBDF) is sponsoring Care Beyond Diagnosis in its efforts to draft international guidelines to…