The phase 1 trial is testing the safety tolerability, pharmacokinetics, and pharmacodynamics of PVT201 in healthy volunteers and patients.

The decision to terminate the HYPERION trial reflects a loss of clinical equipoise, making it unethical to continue the placebo-controlled study.

Patients with ATTR-PN have a high rate of comorbidities and substantial direct healthcare and economic burdens, a study found.

AGEP can closely mimic generalized pustular psoriasis (GPP), requiring histopathologic confirmation and assessment of peripheral eosinophilia.

Associations between therapies and quality of life (QoL) in patients with generalized pustular psoriasis (GPP) can differ substantially.

Daily supplementation of beta-hydroxy-beta-methylbutyrate (HMB) could improve overall daily activity in Duchenne muscular dystrophy (DMD), a study found.

A little more than a third (36.1%) of patients with Huntington disease (HD) and chorea are taking an anti-chorea medication, a study found.

TDP43 seems to worsen the inflammatory activation of astrocytes in neuromyelitis optica spectrum disorder (NMOSD), a study found.

Surgical resection significantly improved overall survival and cancer-specific survival in primary central nervous system lymphoma (PCNSL).

Patients with combined diagnoses of ATTR-CM and aortic stenosis were at an increased risk of hospitalization and death, a study found.

Researchers found that pediatric male patients with hemophilia are at a greater risk of falls due to poor dynamic balance.

As key downstream effectors, PSMB1, CTSD, and CTSH may contribute to BRSK2’s effects on pulmonary fibrosis, with DNA methylation playing a crucial role in its pathogenic mechanism. Results from ...