GCLC-mediated suppression of ferroptosis contributes to lenvatinib resistance in HCC organoids and xenografts. Functional genomic screening revealed that hepatocellular carcinoma (HCC) survival ...

An ongoing open-label extension study suggests that the therapy's benefits may extend through at least 52 weeks. Deupirfenidone (LYT-100), a next-generation antifibrotic therapy, demonstrated a ...

Results showed that 91% of patients had not been diagnosed or even suspected of having CA before death. Cardiac amyloidosis in the forms of transthyretin amyloid cardiomyopathy (ATTR-CM) and ...

Long-term mortality is higher among elderly patients with myasthenia gravis (MG) compared to elderly patients without the disease.

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Herwig Lange, MD, a neurologist who has been researching Huntington disease (HD) since 1969. Dr. Lange, a proponent of nondrug ...

The potential of axonal damage as a biomarker of disease severity and predictor of further outcomes is highlighted by its effect on functional disability, even in early disease stages. Patients who ...

The prevalence of AATD in the offspring of parents with AATD in our study was 37%. A risk of pregnancy complications and adverse birth outcomes has been observed in mothers with alpha-1 antitrypsin ...

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 ...

In this episode of the Rare Care podcast, Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kfir Oved, PhD, about the latest Israeli research on myasthenia gravis (MG). Dr. Oved ...

This comprehensive popPK analysis adds to the pool of knowledge on oligonucleotide PK properties and helps optimize the appropriate use of imetelstat. The nonlinear mixed-effects model identified ...