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 ...

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) ...

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 ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

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, ...

Rituximab is safe and effective in patients with moderate to severe juvenile myasthenia gravis (MG), according to an international cohort and literature review published in the European Journal of ...

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 ...

Survival outcomes were similar with hypomethylating agents (HMAs) alone and an HMA plus venetoclax despite higher overall response rates. Investigators retrospectively evaluated 140 patients with TP53 ...

Key phase 1 interim findings include a half-life of approximately 100 days, which could facilitate once-yearly dosing. Oruka Therapeutics, Inc. has announced positive interim phase 1 results for ...

As we look over the evolution of medicine, we see we have gone from what can be described as crude experimentation in ancient times to more sophisticated ways of managing the signs and symptoms of a ...

A new capsid improves skeletal and cardiac muscle tropism, reducing off-target effects and enabling rapid transduction and transgene expression. New strategies to overcome immune responses against ...