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 diagnosis of small bowel involvement in AAV presents a challenge, with contrast-enhanced abdominal imaging and endoscopic assessment recommended. Patients with antineutrophil cytoplasmic antibody ...

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 ...

BTK has crucial functions in the immune response and is a key component of both the B-cell receptor and Fcγ receptor signaling pathways. Treatment with the Bruton tyrosine kinase (BTK) inhibitor ...

Blincyto ® (blinatumomab), manufactured by Amgen, is a bispecific CD19-directed CD3 T-cell engager. It is approved by the US Food and Drug Administration (FDA) for the treatment of adults and children ...

Between baseline and week 52, spinal canal dimensions numerically improved with navepegritide compared to placebo across L1 to L5. Weekly administration of navepegritide led to improvements in overall ...

The study will assess the number and severity of dose-limiting toxicities and adverse events. A new study will evaluate the safety and efficacy of universal allogeneic anti-CD19/BCMA CAR T-cells in ...

Questions remain regarding the exact relationship between AAV and inflammatory bowel diseases. A young adult female patient with a history of ulcerative colitis was subsequently diagnosed with ...

In patients who had undergone limb lengthening surgery, the annual growth velocity increased from 2.2 ± 0.7 to 6.5 ± 0.7 centimeters per year. Vosoritide significantly increased annual growth velocity ...

The findings from this study highlight the importance of further investigation of nailfold videocapillaroscopy when evaluating of patients with AAV in different age groups. In children with ...

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 ...

The primary outcome measures include assessing I-RODS scores at baseline and monitoring variations at 6 and 12 months after enrollment An upcoming observational, noninterventional study aims to assess ...