Challenges in Management Treatment for AOSD is focused on managing symptoms to prevent complications with the goal of achieving complete remission and preventing joint damage. Although some patients ...

Only 2 disease-modifying idiopathic pulmonary fibrosis (IPF) therapies have been approved by the US Food and Drug Administration (FDA) as of June 2025. They do not stop or cure the disease, so the use ...

A new study described the disease course and outcomes of 5 patients in Kuwait with Gaucher disease type 3c associated with the Asp448His GBA1 variant. Rare Disease Advisor, a trusted source of medical ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews former college basketball player Dan Lier, a partner with the nonprofit group Somebody To Talk To, about his new “Rare Disease ...

Follow Rare Disease Advisor's Gaucher disease junction for the latest news, perspectives, and resources for treating Gaucher disease.

Treatment with porcine antihuman T-lymphocyte globulin (ATG) and cyclosporine, with or without a thrombopoietin receptor agonist (TPO-RA), was effective and generally well tolerated as first-line ...

When you have a rare disease such as cold agglutinin disease (CAD), you need to be diligent to avoid infections. One common infection I have seen over and over being discussed is a urinary tract ...

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare immune-mediated neurological disease characterized by the inflammation of nerve roots and peripheral nerves and destruction of the ...

The prevalence of multiple sclerosis (MS) is increasing globally, particularly in developed countries, according to a study in the Journal of Epidemiology and Global Health. Over the past 30 years, ...

A case of a patient with a rare combination of myelofibrosis (MF) and pyoderma gangrenosum was recently published in the Journal of Cutaneous Immunology and Allergy. “Pyoderma gangrenosum (PG) is a ...

The transactivating response region DNA binding protein 43 (TDP43) seems to worsen the inflammatory activation of astrocytes in neuromyelitis optica spectrum disorder (NMOSD), according to a new study ...