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 ...

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 ...

In observance of International Pompe Day, Rare Disease Advisor has curated this collection of content to illustrate the issues facing the Pompe disease community, highlight the advocacy work being ...

Dentists should be aware that manifestations of hereditary angioedema may be triggered by certain dental procedures. A boy who presented for dental treatment experienced periorbital and facial ...

PHOENIX, Arizona—Biomarkers “are a hot topic in myasthenia gravis (MG),” Anna Punga, MD, PhD, said during her keynote speech at the Myasthenia Gravis Foundation of America (MGFA) session of the 2023 ...

Success rates were highest for HLA-matched units, at 48.3%, followed by cross-matched units at 35.3%, and random units at 22%. A recent retrospective study highlighted the importance of human ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare neurological disorder of peripheral nerves that develops when dysregulated immune mechanisms cause inflammation. Immune-mediated ...

Over 80% achieved a clinically meaningful improvement in at least one of the scales assessed. Treatment with risdiplam over 30 months led to overall clinically meaningful improvement in most adult ...

The patient recovered from surgery with no respiratory complications or any exacerbation in the symptoms of CIDP. Researchers from Iran presented the case of a patient with chronic inflammatory ...

Approximately 31% of dermatologists reported difficulty with flare management, citing slow response to treatment (63%) and lack of flare prevention (66%) as key challenges. There is an urgent need for ...

A new, phase 2b, multicenter, randomized, double-blinded, placebo-controlled, racemate study will evaluate the efficacy and mechanism of action of tezepelumab in adults with eosinophilic ...

Arrowhead Pharmaceuticals announced the first patient treated in Takeda’s phase 3 REDWOOD trial of fazirsiran (TAK-999/ARO-AAT) for patients with alpha-1 antitrypsin deficiency (AATD)-associated liver ...