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.
The study highlights the benefits a robust genetic testing program can have, which ultimately has the potential to improve outcomes ATTRv is caused by more than 130 known mutations in the TTR gene and ...
The consequences of cystic fibrosis (CF) are extensive and affect almost every organ system. The clinical picture is broad and may present with acute or chronic manifestations. Gastrointestinal and ...
Manifestations of chronic inflammatory demyelinating polyneuropathy can be so severe as to significantly impact quality of life. Much ink has been spilled chronicling the disease burden, indeed the ...
Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...
Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...
Between 2001 to 2011, annual age-adjusted incidence increased from 0.55/100,000 to 0.78/100,000, demonstrating an increasing trend with age. Incidence peaks in individuals over 70 years of age ...