Articles related to RARE CONDITIONS & DISEASES

“We are surprised and disappointed by this FDA decision for EBV+ PTLD patients who have a significant unmet need, highlighted by tabelecleucel’s orphan drug designation and by the granting of breakthrough status.” — Dr. Cokey Nguyen, Atara CEO

“Although historically grouped together, viral-associated PCNSL subtypes are biologically distinct diseases. Each escapes immune control through a different mechanism, creating fundamentally different tumor microenvironments.” – Melinda Burgess, Researcher from the Department of Haematology at Princess Alexandra Hospital

Similar efficacy was observed in pediatric patients aged 12 years and older – demonstrating consistent treatment benefit across age groups in the pivotal trial.

Rates increased 3.7% annually with goblet cell tumors nearly five times more common in 1980 births versus 1945 births, suggesting generational risk factors.

SEER registry analysis revealed striking birth cohort effects with appendiceal adenocarcinoma rates more than quadrupling in the 1985 birth cohort compared to the 1945 reference group.

Prior to CDC’s Sickle Cell Data Collection program, the actual number of Americans living with sickle cell disease was unknown, with current estimates of 100,000 patients based on systematic data collection efforts now at risk.