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 ...

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 ...

The 6-minute walk distance corresponded to around 65% of the expected performance in patients with ATTR-CM. Physical performance is “remarkably compromised” in patients with transthyretin-mediated ...

The revisions include the addition of a boxed warning that describes the risk of serious hepatic injury and acute liver failure as well as possible fatal outcomes and the limitation of the use of the ...

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 ...

Patients with FVC below 50% predicted had the poorest outcomes, and within this group, PH and smoking status further stratified prognosis. Pulmonary hypertension (PH) is a key comorbidity among ...

Patients treated with deep brain stimulation of the centromedian nucleus (CM-DBS) showed improvements in seizure burden and alertness as reported by their caregivers. Deep brain stimulation of the ...

Unmanaged symptoms in hemophilia can restrict physical activity and reduce quality of life. Pharmacokinetic-guided treatment personalization allows patients with hemophilia A to become more physically ...

Patients with CIDP and disability had more than 1.8 times the total adjusted all-cause healthcare costs than those without disability, driven by high inpatient costs. Patients with chronic ...

Greater understanding of the pathological effector mechanisms should lead to more rapidly effective and targeted therapies The immunology and neuropathology of autoimmune neuropathies (AIN) that share ...

Lyza Weisman (at right), who has spinal muscular atrophy, with her caregiver, Soraya Perales, at the 2025 MDA conference in Dallas, Texas (Photo by Larry Luxner) Lyza Weisman spoke at the recent MDA ...

Kerrie Walters, who has MS, and her service dog, Wooldridge, at home in Grants Pass, Oregon (Credit: Jennifer Butler Photography) Kerrie Walters first heard of PoNS in a chance encounter with TV ...