US FDA Approves Ctexli, First Treatment for Cerebrotendinous Xanthomatosis of Mirum Pharma
The first medication approved by the FDA to treat CTX, a rare lipid storage condition, is Ctexli.
Intriguing New Class of Antimalarial Drugs Discovered
A study team headed by Gernot Längst (University of Regensburg) and Markus Meißner (LMU Munich) has made significant progress in understanding the gene regulation of the malaria pathogen.
Positive Outcomes From the Initial Prenatal Therapy for Spinal Muscular Atrophy
Researchers at St. Jude Children’s Research Hospital report findings from a novel and promising treatment for the uncommon neurological disease.
Development of Innovative DNA Hydrogels for Assisted Drug Release
Utilizing reduced Takumi-shaped DNA nanostructures, researchers create optimal DNA hydrogels with possible biological uses.
The Risk of Inherited Cancer Can Be Caused by Changes in DNA and Other Biological Pathways
According to the researchers, these recurring patterns suggest potential therapeutic targets meant to prevent or slow the progression of cancer.
Mice Show Promise Results in Gene Therapy for Rare Epilepsy
Rare yet debilitating disorders, Dravet syndrome, and other developing epileptic encephalopathies induce a variety of symptoms in children, such as seizures, intellectual incapacity, and even [Read More…]
Research Finds Out Bacteria Use a Protein Shuttling Mechanism to Discharge Antibiotics
With the mechanism now known, scientists are attempting to sabotage the process with chemical and mechanical means to allow antibiotics to function without hindrance.
Latest Research Finds Out New Blood Clotting Disorder
According to the study, even in the absence of established triggers for these antibodies, such as blood thinners (heparin) or previous vaccinations.
The FDA has approved GOMEKLIâ„¢(mirdametinib) for the treatment of NF1-PN in both adult and pediatric patients, according to SpringWorks Therapeutics
The U.S. Food and Drug Administration (FDA) has approved GOMEKLIâ„¢ (mirdametinib), SpringWorks Therapeutics, Inc.’s MEK inhibitor, for the treatment of adult and pediatric patients with [Read More…]
Search for Novel Tuberculosis Drug Targets Accelerated by Artificial Intelligence
A biotech startup that was spun off from UC San Diego uses deep learning to screen for potential antibiotic candidates as a TB outbreak spreads in Kansas.
