Among patients with severe hemophilia A and B, treatment with the monoclonal antibody marstacimab has been associated with reduced rates of bleeding, according to a systemic review and meta-analysis ...

Sheila Jacobs is the president of STJ Editorial Services. For more than 25 years, Sheila has been providing writing, fact-checking, and editorial services to numerous clients in the medical education, ...

Spesolimab is a therapy that works by targeting interleukin-36 receptor signaling. Its efficacy against generalized pustular psoriasis (GPP) was established in the Effisayil studies; these studies ...

“Andembry, a novel once-monthly subcutaneous treatment that inhibits factor XIIa, is a welcome addition to the HAE treatment landscape,” said Anthony J. Castaldo, CEO and chairman of the board of the ...

Rilzabrutinib, an oral Bruton tyrosine kinase inhibitor (BTKi), demonstrated durable platelet responses, improved physical fatigue, and reduced bleeding in adults with persistent or chronic immune ...

Chronic lymphocytic leukemia (CLL) affects approximately 4.6 per 100,000 individuals per year and is the most common form of leukemia, despite its relative rarity. As the median age of diagnosis is 70 ...

I believe that more people who are living with Pompe disease can walk than those who can’t walk and use a wheelchair. The reason behind why some of us, like myself, aren’t able to walk anymore and ...

AAV.GMU01 SS3-GBA1 (SS3-GBA1), a secretable gene therapy candidate, safely and effectively restored glucocerebrosidase (GCase) function and cleared lipid substrates in rodent and nonhuman primate ...

Which of the statements below about the epidemiology of generalized pustular psoriasis (GPP) are correct? Select one of the options for each question, then submit your answers to see how well you ...

Children with Prader-Willi syndrome (PWS) and Angelman syndrome face significant early health challenges, according to findings from the EUROlinkCAT study recently published in Archives of Disease in ...

Transthyretin amyloidosis (ATTR)-specific treatments and aortic valve replacement (AVR) are associated with significant survival benefits in patients with concomitant aortic stenosis and TTR amyloid ...

Researchers from China synthesized a new caffeic acid borate-derived linear polyglycidol polymer micelle (CAPG) to deliver a small interfering RNA (siRNA) to inflammatory sites in the liver that can ...