Yiannis Papazoglou is one of only 40 people in the world with a mutation of the IRF2BPL gene. Foundation Aims to Help Patients With Rare Diseases—Even Before They’re Recognized The Undiagnosed ...

Top-line results are expected in the first half of 2026, with findings potentially supporting regulatory approval for in IPF. The TETON 1 study, a phase 3 randomized, placebo-controlled trial ...

Sangeetha Venugopal, MD, is assistant professor of clinical medicine at the University of Miami Sylvester Comprehensive Cancer Center. Larry Luxner, senior correspondent for Rare Disease Advisor, ...

Although plasmapheresis showed promise as a treatment to mitigate the risks for severe HDFN, the current study underscored the importance of close monitoring due to potential complications such as ...

A prominent association was demonstrated between the lowest alpha-1 antitrypsin levels and asthma exacerbations. An analysis of DNA samples from individuals with severe asthma and a review of alpha-1 ...

Can Do MS provides free in-person, virtual, and on-demand programs on topics such as nutrition, exercise, MS symptoms, relationships, and employment. Treatment for multiple sclerosis includes much ...

Bioinformatics analysis identified overlapping differentially expressed genes between eosinophilic asthma and EGPA, highlighting shared pathogenic mechanisms. Bioinformatics analysis revealed a ...

Patients with moderate SCD had a 3-fold higher likelihood of abnormal ECG results compared to those with mild disease. Patients with sickle cell disease (SCD), particularly those with severe cases, ...

Close monitoring is vital for managing patients with SRH, a rare but significant complication of AAV. A 52-year-old man presented to the rheumatology department with a 2-week history of anorexia and ...

Sitting height and arm span are useful measurements for patients newly undergoing pharmacologic therapy for short stature, as these enable the monitoring of changes in body proportions. Accurate ...

Kidney transplantation has been shown to be safe and successful in the population of patients with AAV-associated glomerulonephritis and ESKD. Undergoing kidney transplantation is linked to ...

Which of the statements below about the prognosis of chronic lymphocytic leukemia (CLL) are correct? Select one of the options for each question, then submit your answers to see how well you scored ...