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 ...

Therapies for Gaucher disease generally fall into 2 main categories: enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). Neither ERT nor SRT effectively treats the neurological ...

General anesthesia provides similar pain control to nerve block-assisted anesthesia after limb lengthening surgery, common in achondroplasia.

Patients with hepatocellular carcinoma (HCC) face a significantly elevated risk of thrombosis, with incidence higher in Europe.

Are you a health care provider looking for information on sickle cell disease life expectancy? Read more about it here.

Intravenous immunoglobulin therapy, a key treatment used in managing FNAIT, has been linked to the development of thrombocytopenia.

Jakira Avery was diagnosed with limb-girdle muscular dystrophy (LGMD) at a very young age. She uses a wheelchair and is dependent on a ventilator at night due to her having a tracheotomy. Jakira ...

Larry is a freelance journalist and photographer who specializes in covering the rare disease community.

Follow the latest news, insights and perspectives on the treatment of Chronic Inflammatory Demyelinating Polyneuropathy at Rare Disease Advisor.

Bruce is an advocate for Pompe disease. Confined to a wheelchair with complete paralysis, and being told he would never walk again, set Bruce in literal motion to engage in a range of sports and ...

Get the latest news, resources, and perspectives on spinal muscular atrophy (SMA), written specifically for HCPs who treat the disease.

Are you a health care provider inquiring about the signs and symptoms of hemolytic disease of the fetus and newborn (HDFN)? Read more here.