(L-R) Rare Disease Advisor senior correspondent Larry Luxner, patient columnists Alithea Athans, Tara Keith and Tom Bartlett, and director of advocacy relations Vera Luxner (Photo by Riya Ajmera) ...

The study will measure annualized bleeding rate, joint bleeds, spontaneous bleeds, activated partial thromboplastin time, and inhibitor development. A new study is set to evaluate the effectiveness of ...

The tool addressed gaps in exposure to rare bleeding disorders often missing in traditional curricula. An online, case-based learning tool significantly enhanced trainee confidence and perceived ...

Ultrasounds are a relatively cheap and effective tool in monitoring musculoskeletal well-being among patients with hemophilia. There has been a significant rise in the use of ultrasounds to assess ...

Astragaloside II (AS-II) exerted its activity by suppressing β-catenin/inhibitors of DNA binding 2 (Id2)/myelin basic protein (MBP) signaling. Astragaloside II (AS-II) improves neurobehavioral ...

Genetic testing may be a prudent step in the HAE diagnostic process, but clinicians need to be confident of interpreting the results. Hereditary angioedema (HAE) is characterized by unpredictable and ...

This article explores the impact of the EAN/PNS guideline on clinical practice for chronic inflammatory demyelinating polyneuropathy (CIDP). Chronic inflammatory demyelinating polyneuropathy (CIDP) is ...

While NMOSD has been widely studied, limited data exist on the subset associated with autoimmune diseases (AD-NMOSD), particularly in Taiwanese patients. Taiwanese patients with autoimmune ...

Serum levels of α-CGRP are a potential biomarker in NMOSD for sensory and bowel/bladder dysfunction derived from spinal cord lesions. Alpha-calcitonin gene-related peptide (α-CGRP) expression is ...

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 ...

Some 10% to 20% of patients with GPA are negative for ANCA, as occurred in this case. An unusual case has been published in Radiology Case Reports of granulomatosis polyangiitis (GPA), a form of ...

The “Where technology meets human: Redefining patient personalisation” panel at the World Orphan Drug Congress USA 2025 (Photo by Riya Ajmera) Panelists discussed whether AI might reduce the level of ...