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.

While ATTRv-CM may appear in individuals aged in their 50s or 60s, ATTRwt-CM is more common and typically affects men aged over 60 years. The disease is often diagnosed only in advanced stages when ...

The international organizations that have released guidelines for the diagnosis and management of chronic lymphocytic leukemia (CLL) include the National Comprehensive Cancer Network (NCCN), the ...

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 ...

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 ...

Future research in SM will focus on optimizing drug use—perhaps in combinations—safety, and cost-effectiveness, considering limited resources. Increasing the awareness of systemic mastocytosis (SM) ...

When treating MF, clinicians customize treatments to align with each patient’s specific risk profile and disease characteristics. Myelofibrosis (MF), a progressive myeloproliferative neoplasm, is ...