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 ...

Between 21.4% and 57.8% of patients from a real-world ATTR-CM cohort would have been eligible to take part in clinical trials testing disease-modifying therapies. Inclusion and exclusion criteria used ...

The devices are available for patients aged 17 years and older at no extra cost and can be used after receiving training from a healthcare professional. Two new medical devices that will facilitate ...

POEMS syndrome remains a diagnostic challenge due to its variable presentation and overlap with other demyelinating neuropathies, such as CIDP. Cases of polyneuropathy, organomegaly, endocrinopathy, ...

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 majority (90%) of neurologists were aware of 40 out of 61 recommendations on the treatment of myasthenia gravis (MG). Most Spanish neurologists know, agree on, and apply guidelines for the ...

Intercept Pharmaceuticals, the developer of the PBC drug, suggested that the company's view on the drug's benefit and risk profile differed from that of the US Food and Drug Administration.

In this episode of the Rare Care podcast, Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Kfir Oved, PhD, about the latest Israeli research on myasthenia gravis (MG). Dr. Oved ...

In addition to a 69.4% CR rate, the team observed good tolerability of the R2-MTX regimen, suggesting its suitability for older or less fit patients. High-dose methotrexate (HD-MTX) with lenalidomide ...

Jerry Lewis, one of the most famous comedians of the 20th century, was a tireless advocate for people with muscular dystrophy. Larry Luxner, senior correspondent for Rare Disease Advisor, interviews ...

A low ALBI score reflected better liver function and predicted improved OS and RFS. The albumin-bilirubin (ALBI) score can independently predict prognosis in post-surgical patients with distal ...

(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) ...