FDA approves 1st treatment for Menkes disease, offering a copper therapy that improves survival in affected children.
Berkeley health officials are urging people to vacate a homeless encampment after an outbreak of a rare disease that can ...
Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...
Kawasaki Disease is a rare heart condition that usually affects children under the age of 5. According to Johns Hopkins ...
Torie Bosch is the First Opinion editor at STAT. Celena Lozano’s son Benny, who turned 5 in November, loves trains, trucks — anything that goes. He also has a rare disease, PURA syndrome. Earlier this ...
The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.
The Republic of Serbia allocates millions for the treatment of rare diseases, and in 2025 alone, more than 930 patients in ...
Danish dermatology specialist Leo Pharma and its CEO Christophe Bourdon came to San Francisco to attend the J.P. Morgan ...
Caregivers and families reflect on life after loss, as the Star PALS team remembers patients past, and parents continue ...
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