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 ...

Long-term safety was comparable to standard of care, with no new safety signals identified over 52 weeks. CAM2029, a long-acting octreotide subcutaneous depot, provided sustained biochemical control, ...

The use of semaglutide has been associated with improvement of treatment-resistant mast cell activation symptoms in an overweight patient with SM. Glucagon-like peptide-1 receptor agonists (GLP-1RAs) ...

Taylor Kane is executive director of Remember the Girls, a nonprofit group that advocates on behalf of females with X-linked disorders (Photo courtesy of Taylor Kane) New Jersey native Taylor Kane ...

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 ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Jennifer Miller, MD, a professor of pediatric endocrinology at the University of Florida in Gainesville who specializes in ...

CAD accounts for approximately 15% of AIHA cases 2; various sources indicate that anywhere from 7% to 25% of cases of AIHA are caused by CAD. 3 Secondary CAD occurs as a result of underlying ...

In this case, avacopan monotherapy yielded a sustained AAV remission lasting for over 6 months, even in the absence of steroid treatment. Avacopan can induce sustained, steroid-free remission in ...

Dimitrios Karussis, MD, PhD, head of the Multiple Sclerosis Center at Hadassah University Hospital in Jerusalem, Israel (Photo by Larry Luxner) Patients with multiple sclerosis who were injected with ...

All NORD-designated Centers of Excellence meet rigorous benchmarks for multidisciplinary expertise, patient education, clinician training, and active engagement in research. The National Organization ...

Dana Rizk, MD, professor of medicine in nephrology at the University of Alabama at Birmingham Heersink School of Medicine, speaks at the Renal Physicians Association 2026 Annual Meeting in Atlanta, ...

The Stride Velocity 95th Centile (SV95C) showed sensitivity to a decline in the ability to walk over short intervals in patients with Duchenne muscular dystrophy (DMD), according to a new study ...