The US Food and Drug Administration (FDA) approved Panzyga, manufactured by Octapharma AG, for the treatment of PI and ITP in 2018 and for CIDP in adults in 2021. Octapharma commercializes Panzyga ...

Dan Lier has light chain (AL) amyloidosis—the most common form of amyloidosis. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews former college basketball player Dan Lier, a ...

The prognostic model for NMOSD-optic neuritis risk score included female gender, optic neuritis in both eyes, the absence of pain, and chiasmal involvement detected in orbital magnetic resonance ...

The Muldoon family from Philadelphia, Pennsylvania, is shown after baby KJ’s infusion of a personalized gene-editing therapy (Photo courtesy of Children’s Hospital of Philadelphia) KJ Muldoon has been ...

The results showed that SGLT2i therapy significantly reduced all-cause mortality, in-hospital mortality, and cardiovascular mortality. Sodium-glucose cotransporter-2 inhibitors (SGLT2is) appear to ...

The growth rate for girls was 5.8 centimeters per year with vosoritide compared with 4.2 centimeters per year without treatment. After the onset of puberty, vosoritide induced an accelerated growth ...

Anti-FVIII antibody profiles are closely linked to inhibitor levels in cases of acquired hemophilia A. An exercise in antifactor VIII (FVIII) antibody profiling showed distinct patterns in acquired ...

Treatment with inebilizumab led to higher rates of flare-free, treatment-free remission and a significant reduction in steroid use. Inebilizumab, a CD19-targeting monoclonal antibody, significantly ...

Using a cutoff value of 1.87 µmol/L, CuEXC achieved a sensitivity of 76.9% and a specificity of 99.6% in differentiating newly diagnosed WD from non-Wilsonian hepatic disease. Relative exchangeable ...

For the last 3 years, the PBC Foundation, based in Edinburgh, Scotland, has conducted an annual summit that addresses the challenges and potential remedies for those living with primary biliary ...

Efforts to further risk stratify patients with cardiac amyloidosis who may benefit from ICD implantation are needed. No significant difference in mortality was observed between patients with ...

Today was a long day. I normally write about living with cold agglutinin disease (CAD), though I contemplated not writing this week. But then I remembered that May is National Stroke Awareness Month.