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.

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

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.

Although a biopsy is essential to diagnose IgG4-RD and AAV, in those cases in which a biopsy cannot be performed, clinical features and supportive imaging or laboratory data may provide important ...

Patients with GPP who experienced flares had higher hospitalization rates (12.2% vs 6.9%) compared to those who did not flare. Patients with generalized pustular psoriasis (GPP) who experience flares ...

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

Iptacopan shows promise in reducing fatigue and improving function in patients with PNH. Iptacopan resulted in meaningful improvements across a number of key clinical parameters in patients with ...

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.

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

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