(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 ...

In CIDP, SOC therapies (immunoglobulins/corticosteroids) are burdensome, have variable efficacy, and significant side-effects. Two phase 3 clinical trials, MOBILIZE ...

The patient with NMOSD presented with vision loss in his right eye 9 years after his initial diagnosis. Progressive multifocal leukoencephalopathy (PML) can occur in patients with childhood-onset ...

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 ...

Due to the close temporal relationship between intravenous immunoglobulin administration and the onset of conduction abnormalities, there may be a potential causal association between the 2 events.

While I was sick with chronic inflammatory demyelinating polyneuropathy (CIDP), but before my diagnosis, my lack of sensation and proprioception had left me crawling around my apartment, wall walking, ...

Sara Riordan, MS, vice president of strategy at Genome Medical (left) and Natalie M Beck, MGC, senior manager of clinical trials and real-world data at Genome Medical speak at the 2025 World Orphan ...

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 ...

Michael Lingzhi Li, PhD, Assistant professor in the technology and operations management unit at Harvard Business School, Boston, Massachusetts, speaks at WODC 2025 (Photo by Riya Ajmera) Speakers ...