Coramitug is well tolerated and leads to a statistically significant reduction in N-terminal pro-brain type natriuretic peptide (NT-proBNP)—a marker of disease progression in transthyretin-mediated ...

The mean cost savings associated with tafamidis treatment due to a reduction in hospitalization because of heart failure and shortening of length of stay in the hospital was $12,940 per patient.

The ratio of posterior wall thickness to the minimum voltage of QRS complexes in limb leads and several parameters derived from electrocardiography could independently predict ATTR-CM. The ratio of ...

Diagnosis of neurofibromatosis type 1 (NF1) requires a combination of clinical assessment, genetic testing, and imaging studies. 1 NF1 is a rare genetic disorder characterized by the development 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 ...

Emerging therapies such as bezuclastinib and elenestinib show promise in improving disease control with potentially fewer toxicities. Other novel strategies, such as immune-based therapies, are under ...

The potential of axonal damage as a biomarker of disease severity and predictor of further outcomes is highlighted by its effect on functional disability, even in early disease stages. Patients who ...

(L-R) Rare Disease Advisor senior correspondent Larry Luxner, patient columnists Alithea Athans, Tara Keith and Tom Bartlett, and director of advocacy relations Vera Luxner (Photo by Riya Ajmera) ...

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, ...

Patients with ANCA-positive interstitial lung disease (ILD) exhibited a high prevalence of fibrotic patterns and mortality largely driven by the ILD itself. In patients with interstitial lung disease ...

Obeticholic acid, seladelpar, and elafibranor all significantly increased the biochemical response rate. Elafibranor seems to be slightly more effective than seladelpar as a second-line therapy in ...

With the approval of the indication extension, pegcetacoplan now stands as the first C3 inhibitor for first-line PNH treatment. The European Commission (EC) has approved the indication extension of ...