OTS

Growing up the oldest of four children in Leiden, Netherlands, Dr. Annemieke Aartsma-Rus became fascinated with medical research and genetics as a teenager. While her father's work as a physician made her realize she didn't want to be a doctor, it did teach her ...

When Yessenia Gutierrez was just nine years old, her kidneys failed; at 10 years old, she received her first kidney transplant. Gutierrez was born with polycystic kidney disease (PKD), a genetic condition that makes fluid-filled cysts grow on the kidney and can lead to ...

In 2024, the Food and Drug Administration Center for Drug Evaluation (CDER) approved 50 new small molecules, biologics, and oligonucleotide therapies (1). The approvals — the second largest in 30 years — include an oligonucleotide therapy for blood cancer, an antisense oligonucleotide shown to ...

Antisense oligonucleotide (ASO) therapies, which bind to RNA and modify protein expression, are promising drugs for treating neurological conditions. However, their inability to cross the blood-brain barrier (BBB) sometimes makes injection directly into the cerebral spinal fluid (via intrathecal injection) necessary, which is not ...

At just three weeks old, Darlene was sent to the emergency room after her mom had brought her to a pediatrician. The baby wouldn't stop crying and wasn't eating. Concerningly, her triglyceride levels were in the 20,000's, far over the normal 150 level. Darlene ...