On June 14, 2021, when people wished each other a "good Dragon Boat Festival", a patient with paroxysmal nocturnal hemoglobinuria (PNH), a rare disease of hematology, waited anxiously for 83 days and finally ushered in her new life. The world's leading Phase III clinical medications were sent to Peking Union Medical College Hospital from Switzerland as an expanded compassionate drug use. Representatives from Peking Union Medical College Hospital, National Medical Products Administration, China Rare Diseases Alliance, and Novartis China gathered together to hold a simple and solemn donation ceremony of compassionate drug use for the patient.

The first case of compassionate drug use for rare diseases in China was successfully implemented in PUMCH.

PUMCH clinical and management MDT meeting was held on April 28th at the Difficult Disease Consultation Center. More than 30 clinical and management experts discussed the treatment of PNH patients.

Donation ceremony of compassionate drug use for rare diseases was held in PUMCH on June 14th.

Reporter: Chen Mingyan, Wang Jing

Photo：Sun Liang

Translator: Pan Qingqing

Editor: Xu Xiaohan