The global, double-blinded phase 3 study enrolled 165 patients aged between 12 and 75 years and measured the time to onset ...
FDA approves 1st treatment for Menkes disease, offering a copper therapy that improves survival in affected children.
Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...
A 2-year-old boy died from a rare disease with flu-like symptoms, just weeks before his third birthday. Hudson Hughie Martin, ...
Danish dermatology specialist Leo Pharma and its CEO Christophe Bourdon came to San Francisco to attend the J.P. Morgan ...
Rare diseases are genetic disorders that affect a small proportion of the population. Despite their rarity, they can be a ...
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 ...
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 ...
After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told ...
The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.
Caregivers and families reflect on life after loss, as the Star PALS team remembers patients past, and parents continue ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback