Only 4.6% of our included patients had findings corresponding with disease progression in the chest without any progression intra or extra-hepatic on follow-up CT. Computed tomography (CT) scans of ...

Effective fetal neurological counseling requires acknowledging prognostic uncertainty and delivering precise, patient-centered communication. Effective fetal neurological counseling requires ...

NIPT results can be difficult to interpret due to chromosomal aberrations present in the mother’s malignant cells. Caution is warranted when using noninvasive prenatal testing (NIPT) in pregnant women ...

This review aims to serve as a practical guide for providers seeking to familiarize themselves with neuromodulation devices and techniques for treating patients with LGS. Several different forms of ...

Researchers used artificial intelligence technology to assess its ability to detect PNH in a large data set. Artificial intelligence (AI) technology has accelerated in recent years, and with it has ...

The use of digital patient-reported clinical tracking technology represents an opportunity to significantly enhance hemophilia care. In Therapeutic Advances in Hematology, Ettingshausen and colleagues ...

Surgery is not often used as a treatment option for chronic lymphocytic leukemia (CLL) because the disease is usually widely spread in the bone marrow and other organs. However, surgery may sometimes ...

Overall, 80.9% of patients were in complete remission 1 year after diagnosis irrespective of GPA or MPA cases (79.7% vs 83.8%, respectively). The therapeutic armamentarium for individuals with ...

Vosoritide was approved in 2021, after current achondroplasia guidelines were published, creating a need for treatment and monitoring recommendations. A new international consensus statement has been ...

The navenibart phase 3 program will consist of the ALPHA-ORBIT phase 3 trial and the long-term extension trial, which are designed to support registration globally. Global start-up activities ...

Nusinersen, an SMN2-targeted antisense oligonucleotide drug administered by intrathecal injection, was able to improve motor function and survival. Treatment with nusinersen can improve motor function ...

Dr. Bichell is part of a panel studying how the US FDA can speed up clinical trials and be more transparent about its decisions. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...