Results showed that 91% of patients had not been diagnosed or even suspected of having CA before death. Cardiac amyloidosis in the forms of transthyretin amyloid cardiomyopathy (ATTR-CM) and ...

The study highlights the benefits a robust genetic testing program can have, which ultimately has the potential to improve outcomes ATTRv is caused by more than 130 known mutations in the TTR gene and ...

PNI is an independent protective factor for recompensation in patients with decompensated PBC. Higher scores on the prognostic nutritional index (PNI) appear to be directly correlated with better ...

The levels of NT-proBNP, left ventricular mass index, maximal wall thickness, and E/E′ ratio were higher in patients with worse neurological stages compared to those in the early neurological stages ...

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 ...

Teclistamab and strategies targeting B cell maturation antigen (BCMA) may have therapeutic potential in myasthenia gravis (MG), according to a new case study published in the peer-reviewed scientific ...

No patient in the 6-month rituximab group relapsed during the 24-month follow-up, and 1 patient in the 12-month group had a minor relapse. Tailored, risk-stratified rituximab regimens including yearly ...

C-KIT D816Y mutation and spindle-shaped CD25+, CD2+, CD30+ mast cells confirmed SM with associated hematologic neoplasm, warranting altered treatment strategy. This case demonstrated that without ...

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 ...

Lauren Holder, who has Huntington disease, shows off her “Cure HD” tattoo during the 2025 World Orphan Drug Congress in Boston, Massachusetts (Photo by Larry Luxner) Lauren Holder, 39, was diagnosed ...

Ruxolitinib exerts its activity independently of oncogenic Janus kinase 2 (JAK2)-V617F signaling in myeloproliferative neoplasms (MPNs) like myelofibrosis (MF), according to a new study published in ...

More than half (52.4%) of the patients had target joints, with the mean number per patient ranging from 1.3 to 2.3, irrespective of hemophilia type or inhibitor status. In general, patients receiving ...