HAE affects approximately 1 in 50,000 people globally, but is far more prevalent in a few select geographic regions.

Diazoxide choline is believed to activate the ATP-sensitive potassium channel, which may decrease secretion of peptides that regulate appetite.

The GBS/CIDP Foundation International aims to provide support, with an emphasis on patients and caregivers meeting each other.

Newborn screening in the US currently identifies about 14,000 babies with serious conditions annually.

Lily Sander has an especially rare type of Charcot-Marie-Tooth (CMT) disease—a mutation that occurs in just 1% of those with CMT.

Increasingly, modern medicine is making room for patients to better express themselves through self-reported outcomes, especially for rare diseases like SM.

Studies suggest that rituximab may have a significant role to play in patients who have been diagnosed with refractory CIDP.

Significant gaps remain in the clinical management of generalized pustular psoriasis (GPP), including guidelines on long-term chronic care.

Researchers investigated the apparent close relationship between IGG4-RD and AAV and whether they arise from shared pathophysiological pathways. 

Fibrates was used as second-line therapy in a pregnant patient with PBC-associated pruritus who responded poorly to UDCA, a study found.