Being a primary biliary cholangitis (PBC) advocate is a hard job. It comes with a lot of interpersonal battles as well as challenges I never expected to encounter. Although my advocacy journey feels ...

Which of the statements below about the therapeutic study on Centella asiatica in transthyretin amyloid polyneuropathy (ATTR-PN) are correct? Select one of the options for each question, then submit ...

The 10-year estimated risk of the development of ATTR-CM in family members of patients with the disease is high at 29.5%, a study found.

Anxiety reduction was greater with sebetralstat than placebo at both 4 and 12 hours. Results from the phase 3 KONFIDENT clinical trial indicate that sebetralstat, an oral plasma kallikrein inhibitor, ...

The New York Heart Association class improved or remained stable for 77.6% of patients treated with tafamidis as part of an early access program.

During treatment with nusinersen there was a mean reduction in performance fatigability of 5.6%. Performance fatigability is an independent component of motor impairment and, therefore, a potential ...

NfL levels were evidently correlated with compound muscle action potential (CMAP) and sensory nerve action potential (SNAP) amplitudes. Nerve conduction studies could be a representation of ...

The incidence of positive ATTR deposition among patients who had lumbar spinal surgery was 48 %. Transthyretin amyloid (ATTR) deposits are often found in older patients with spinal stenosis, according ...

Rare disease advocacy groups praised the House’s passage of legislation that extends the Rare Pediatric Disease Priority Review Voucher program until 2029. The Rare Pediatric Disease Priority Review ...

The toxic effect of the TTR amyloid aggregates was reduced in a dose-dependent manner with C. asiatica extract treatment. To explore whether the hydrophilic extract of C. asiatica could suppress the ...

Brain volume loss is not yet a core outcome in relapsing MS clinical trials. However, it is gaining attention due to its correlation with disability progression. Recent findings highlight the value of ...

Philadelphia chromosome positive ALL had superior outcomes following hematopoietic cell transplantation in first complete remission. Hematopoietic cell transplantation, combined with effective ...