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 ...

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, ...

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 ...

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 ...

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 ...

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 ...

In the ECHELON-3 study, the brentuximab vedotin combination with lenalidomide and rituximab reduced mortality risk by 37% compared with placebo. Brentuximab vedotin (Adcetris®) has been approved by ...

Future studies involving indolent SM should include diverse populations to better capture epidemiological differences. Non-White patients may experience a higher disease burden associated with ...

Over my 4 decades living with multiple sclerosis (MS), I’ve had dozens of magnetic resonance imaging (MRI) exams. Almost all of them were done using a contrast agent (often called a dye) containing ...

Which of the statements below about the prognosis of immunoglobulin G4-related disease (IgG4-RD) are correct? Select one of the options for each question, then submit your answers to see how well you ...

With only a week to go until Rare Disease Day 2025, the federal government has shut down its flagship Feb. 27-28, 2025, event in Washington, DC—sparking bewilderment and outrage from national ...

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 ...