Sharon Hesterlee, PhD, who previously served as chief research officer at the MDA, has been named interim president and CEO. Donald S. Wood, PhD, has retired from the Muscular Dystrophy Association ...

A new phase 2 clinical trial to test the safety, efficacy, and tolerability of TAK-411 for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) is now open. The open-label, ...

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 ...

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 ...

The timing of intraventricular hemorrhage (IVH) onset has shifted. IVH occurring before 6 hours of life decreased from 35% before 2007 to 9% after 2007. Intraventricular hemorrhage (IVH), a subtype of ...

Gastrointestinal involvement in systemic mastocytosis (SM) is a common complication and often leads to endoscopic findings. Gastrointestinal (GI) symptoms are the second most frequent clinical ...

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) ...

Although treatment was started with rituximab upon the CAD diagnosis, she died within 5 days. A case has been reported of a patient with a history of Raynaud’s phenomenon who was hospitalized due to ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...

Older patients and those with genetic risk factors were at particularly high risk of relapse and had poorer overall survival. Chimeric antigen receptor-T (CAR-T) cell therapy followed by allogeneic ...

Real-world data confirmed the safety and efficacy of vosoritide, showing height increases of up to 11.86 cm over 24 months in children with achondroplasia. Vosoritide (Voxzogo ®) continued to ...

Speakers at the panel “Unlocking the future: the convergence of AI, personalized medicine, and genomics” at the World Orphan Drug Congress USA 2025 (Photo by Riya Ajmera) Artificial intelligence could ...