A diagnosis of ATTR-CM in a more recent era was associated with older age, White race, wild‐type disease, and tafamidis treatment. The survival of patients diagnosed with transthyretin-mediated ...

Generalized pustular psoriasis (GPP) is a rare, severe form of psoriasis characterized by the acute onset of eruptions of sterile pus-filled skin lesions and rashes with or without systemic ...

Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Herwig Lange, MD, a neurologist who has been researching Huntington disease (HD) since 1969. Dr. Lange, a proponent of nondrug ...

A new phase 2 clinical trial to test the safety, efficacy, and tolerability of TAK-411 for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) is now open. The open-label, ...

Supplemental oxygen therapy, supplied through tanks or an oxygen concentrator, plays a major role in the quality of life of patients with low oxygen levels due to pulmonary arterial hypertension (PAH) ...

From landmark gene therapy approvals to illuminating discoveries into the pathogenesis of rare diseases, 2023 saw substantive advances in rare disease diagnosis and treatment. The following list of ...

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 ...

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

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Albert Faro, MD, vice president for clinical affairs at the Cystic Fibrosis Foundation (CFF). Dr. Faro speaks about COVID-19’s ...

Several factors can contribute to the suppression of bone marrow erythropoiesis in infants with HDFN, including maternal antibodies, intrauterine transfusion, and simple transfusion. The suppression ...

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 ...