Author Archives: Alice Melao

Alpha-Synuclein Protein May Contribute Toward Batten Disease Progression

High levels of a protein associated with Parkinson’s disease, called alpha-synuclein, may contribute to the progression of late infantile Batten disease, according to a recent study. The study, “Autophagy–lysosome pathway alterations and alpha-synuclein up-regulation in the subtype of neuronal ceroid lipofuscinosis, CLN5 disease,” was published in…

Amicus Therapeutics’ Expanded Portfolio Includes Potential Gene Therapies for Batten Disease

Amicus Therapeutics has gained global development and commercial rights to 10 gene therapy programs for lysosomal storage disorders, including three potential first-to-market therapies for Batten disease. This pipeline expansion resulted from the acquisition of Celenex, a clinical stage gene therapy company. “The in-licensing and acquisition of these gene therapy programs…

ABO-202 Earns EMA’s Orphan Drug Status for Infantile Batten Disease

ABO-202, Abeona Therapeutics‘ investigational candidate for the treatment of infantile Batten disease has received orphan drug status by the European Medicines Agency. This new status adds to the previously granted orphan drug and rare pediatric disease designation by the U.S. Food and Drug Administration. These designations are…

Early Detection of Retinal Dysfunction May Help Diagnosis

Early detection of abnormalities to the eye’s retina and loss of visual acuity may help diagnose neuronal ceroid lipofuscinosis (NCL), aka Batten disease, and improve patient care, Brazilian researchers suggest. For several neurodegenerative disorders that have symptom onset during early infancy is not uncommon for the children to develop eye…