DNAtrix will report data from an ongoing Phase 1 trial of DNX-2401 in pediatric patients with newly diagnosed diffuse intrinsic pontine gliomas (DIPG), which indicated efficacy through a prolonged survival rate.
Alnylam Pharmaceuticals has achieved delivery of novel small interfering RNA (siRNA) conjugates to the central nervous system and intends to advance a pipeline of investigational RNAi therapeutics into clinical development.
A human clinical trial grant totaling $750,000 was awarded this morning by the Muscular Dystrophy Association (MDA) to Massachusetts General Hospital, with the hope that it will accelerate biomarker research for amyotrophic lateral sclerosis (ALS).
Bruce Wang, MD, a porphyrias specialist and assistant professor in the gastroenterology division in the department of medicine at University of California San Francisco (UCSF) and UCSF Porphyria Center and the Porphyrias Consortium, explains acute hepatic porphyrias (AHPs).
According to new researche, a widely-used cholesterol-lowering drug increased a precursor of high-density lipoprotein (HDL) in models that mimicked liver cells of patients with Friedrich’s ataxia (FA).
As stated in article published in the clinical journal Neurobiology of Aging, a team of researchers has discovered that elevated levels of certain types of lipids in the brain could be an early presentation of Parkinson’s disease.