There was a reduction in volume of involvement, cardiac pyrophosphate activity, native T1, and extracellular volume during a median of 369 days between scans in patients with ATTR-CM being treated.

A multidisciplinary approach is necessary for the deep phenotyping and proper clinical management of individuals with primary mitochondrial diseases. A large percentage of patients (60%) with primary ...

A pattern recognition approach takes into account factors like disease progression rate, whether the disease symptoms are symmetrical or asymmetrical, whether they are predominantly distal or proximal ...

Getty Images A retrospective analysis of patient health records found that transthyretin stabilizers show clinical benefit in adults with ATTR-CM. Those taking TTR stabilizers had a lower risk of ...

Getty Images A post-hoc analysis of the ATTRibute-CM trial showed that acoramidis significantly improves survival in patients with p.V142I ATTRv-CM. By Month 30, the risk of combined all-cause ...

Denecimig aims to help the body form blood clots more effectively by mimicking the role of FVIIIa and bridging factor IXa and factor X to restore thrombin generation and support normal clot formation.

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 ...

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 ...

The results of the current meta-analysis reinforce the safety and efficacy of marstacimab in reducing annualized bleeding rates among patients with severe hemophilia A and B. Among patients with ...

The updated 2025 BSR management recommendations for patients with AAV provide systematic and evidence-based recommendations to support UK clinicians in managing AAV across the entire life course.

A baseline bleeding risk of 7.3 bleeds per year was observed without any FIX, and targeting trough FIX levels between 10 and 20 IU/dL reduced bleeds to very low or eliminated them. In people with ...

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 ...