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.

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 ...

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 ...

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 ...

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 ...

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 ...

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

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 ...