New leukaemia treatment could reduce need for chemotherapy

New leukaemia treatment could reduce need for chemotherapy in ‘major step forward’ - Doctors have found a gentler treatment ...
Nature

Lysosomal ceroid depletion by drugs: Therapeutic implications for a hereditary neurodegenerative disease of childhood

Neuronal ceroid lipofuscinoses (NCLs) are the most common hereditary neurodegenerative diseases of childhood. The infantile form, INCL, is caused by lysosomal palmitoyl-protein thioesterase (PPT) ...
SurvivorNet

Teen’s ‘Muscular Pain’ In Lower Back & Weight Loss Dismissed Until Blood Test Revealed Acute Lymphoblastic Leukemia (ALL)

After months of dismissed back pain and weight loss, 16-year-old Kit Chester-Canavan, from the U.K., was diagnosed with acute lymphoblastic leukemia when his mother pushed for further testing. Acute ...
Nature

Repeat instability as the basis for human diseases and as a potential target for therapy

In this article, we describe recent findings in disease-associated repeat instability, including the growing importance of tissue and developmental specificity, new model systems that better mirror ...