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

In 2025, the company plans to further their SM program by presenting additional results from the PIONEER trial and activating the phase 3 study of elenestinib for indolent SM. Blueprint Medicines ...

Analysis of genetic similarities between IPF and GERD, 2 conditions that often coexist, may aid in the selection of therapeutic targets. Nine hub genes have been identified as significantly altered in ...

Vamorolone at a dose of 2 mg/kg a day was associated with improvements in the velocity of the 4-stair climb test and the supine-to-stand test. A new systematic review and meta-analysis of the ...

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

Noninvasive prenatal testing has become an essential tool for managing alloimmunized pregnancies. “Despite challenges such as low cff-DNA [cell-free fetal DNA] fractions and complex genetic variations ...

ATTR-PN diagnosis requires a combination of neurological and non-neurological tests, such as blood pressure monitoring, a ...

The findings from this study build on existing literature by demonstrating the value of GPT-3.5 in recognizing a rare condition when provided with clinical information. ChatGPT was able to correctly ...

This case report suggests that onasemnogene abeparvovec could be both feasible and safe in neonates with a history of HDFN. A 21-year-old woman gave birth to a male neonate following a full-term ...

In addition to improved functional AAT levels in the lungs, inhaled AAT demonstrated good safety and tolerability. Patients with alpha-1 antitrypsin deficiency (AATD) receiving inhaled ATT therapy ...

Combined anti-gp210 and anti-centromeric antibody positivity is a risk indicator independent of the GLOBE score and measurements of liver stiffness. Combined anti-gp210 and anti-centromeric antibody ...

Environmental pollution, especially particulate matter 2.5 (PM2.5) has a significant impact on disease severity in patients with multiple sclerosis (MS) and neuromyelitis optica spectrum disorder ...