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 ...

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 ...

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 ...

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 ...