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

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.

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

Chronic lymphocytic leukemia (CLL) clinical features include symptoms seen at presentation, along with features seen due to lymphoid involvement, complications, and extramedullary manifestations.

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

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

Even after obtaining an accurate diagnosis, patients reported experiencing between 1 and 20 attacks per year. Patients with hereditary angioedema (HAE) from underrepresented racial and ethnic groups ...

Children averaged an annual growth velocity of 6.0 cm per year and an absolute height gain of 6.2 cm after 12 months of treatment. In children with achondroplasia in real-world settings, vosoritide ...