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.

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

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

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

The mean maximal mouth opening was 24.5 mm in children with SMA with no teeth and 25.66 mm in those with teeth, which represents an increase with age. Maximal mouth opening is significantly wider in ...

Castleman disease and IgG4-RD have overlapping disease manifestations that can complicate the diagnostic process. A male patient initially suspected of having immunoglobulin-G4 related disease ...

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

Several clinical trials are currently ongoing to improve the treatment of patients with chronic lymphocytic leukemia (CLL). These CLL clinical trials can be grouped based on the type of therapy, ...

Persistent benefits were observed in patients with FA who received omaveloxolone for an extended period. Omaveloxolone seems to lead to persistent benefits in patients with Friedreich ataxia (FA), ...