Intercept Pharmaceuticals, the developer of the PBC drug, suggested that the company's view on the drug's benefit and risk profile differed from that of the US Food and Drug Administration.

Results support incorporating discussions of adverse events alongside efficacy in shared treatment decision-making. Progression-free survival (PFS) and the impact of adverse events (AEs) on quality of ...

Anti-transferrin receptor (TfR) antibody-coupled acid-alpha-glucosidase (GAA) led to robust glycogen clearance and reduced neuroinflammation in the central nervous system of a mouse model of Pompe ...

Relatives who underwent screening were diagnosed and began treatment significantly earlier than the proband by more than a decade. Systematic family screening through genetic testing plays a critical ...

Patients with AATD had a 1.6-fold higher risk of any type of middle ear surgery, even after adjusting for age, sex, COPD, and liver cirrhosis. Patients with alpha-1 antitrypsin deficiency (AATD) are ...

The highest risks were observed for bladder cancer, leukemia, nonmelanoma skin cancer, and kidney cancer. Patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) have a ...

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 ...

The primary goals of AI personalization were to improve onboarding, boost medication initiation and adherence, and enhance physician follow-up. AllazoHealth’s artificial intelligence (AI)-powered ...

Several factors can contribute to the suppression of bone marrow erythropoiesis in infants with HDFN, including maternal antibodies, intrauterine transfusion, and simple transfusion. The suppression ...

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 ...

My recent liver biopsy was a case study of Murphy’s Law in action. And as I share the details of my saga, I’m mindful that my experience is the exception, not the rule. I do not want to discourage ...

Finding a treatment that can restore myelin is my holy grail of multiple sclerosis (MS) research. Will a neurostimulator designed by SetPoint Medical someday be the treatment I’ve been searching for?