Fingolimod treatment leads to beneficial effects in Huntington disease (HD), according to a new study in a mouse model of the ...

Alpha-1 antitrypsin deficiency (AATD) is found to be associated with pregnancy-related complications and adverse birth ...

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 ...

Overall, the life expectancy of patients with SCD is lower than that of persons without SCD. 1 However, life expectancy varies depending on the type of disease, treatments administered, and ...

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 ...

There were fewer multiorgan transplants in patients with hereditary ATTR-CM with increased use of disease-modifying therapies, a study found.

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 ...