The global, double-blinded phase 3 study enrolled 165 patients aged between 12 and 75 years and measured the time to onset ...
Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...
FDA approves 1st treatment for Menkes disease, offering a copper therapy that improves survival in affected children.
Rare diseases are genetic disorders that affect a small proportion of the population. Despite their rarity, they can be a ...
A 2-year-old boy died from a rare disease with flu-like symptoms, just weeks before his third birthday. Hudson Hughie Martin, ...
The seven-year medical odyssey that once defined rare disease diagnosis is being rewritten by algorithms capable of identifying medical zebras in the time it takes to refresh your social media feed.
The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.
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 ...
More than 500 European patients with previously undiagnosed rare disorders received a genetic diagnosis through research carried out by an extensive European collaboration, the Solve-Rare Diseases ...
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 ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback