The stability of lysosomal proteins when tested in a laboratory setting is not equivalent to that observed in live cells,…
Articles by Ana Pena, PhD
New research has deepened the understanding of the underlying causes of Batten disease, including organs and cell types affected…
Neurologists and researchers have tailor-made an antisense oligonucleotide therapy (ASO) for a 6-year-old girl who has CLN7 Batten disease,…
Extracellular vesicles derived from a type of immune cell called macrophage may hold potential as a less-invasive delivery…
Neurogene is sponsoring a natural history study to better understand the course of CLN5 and CLN7 disease,…
Amicus Therapeutics entered a collaboration with Paragon Gene Therapy, a unit of Catalent Biologics, for the production…
A new dried blood spot test may be a reliable and rapid method for obtaining an initial…
Healx has raised $10 million to accelerate therapy discovery for rare diseases, including Batten disease. The…
A group of experts has created a set of guidelines to best manage late infantile Batten disease,…
Investigational therapies now in development — especially gene and cell therapies — are reason for fresh hope among …