Polaryx Therapeutics’ investigative drug PLX-100 has been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration…
Articles by Alice Melao
Researchers studying the loss of the CLN5 protein, which causes Batten disease, found that it leads to the reduced production…
Abeona Therapeutics‘ ABO-201 reduced nerve inflammation and improved movement in mice with juvenile Batten disease (JBD), according to a preclinical trial…