Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging ...
Jaguar has received the initial $16M payment related to the company's recently executed US out-license agreement for ...
A renewal of the FDA’s pediatric rare disease voucher program is couched within a massive $1.2 trillion spending bill the ...
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...
Backed with $82 million and led by two of BioMarin’s former executives, Mendra plans to acquire rare disease assets and use ...
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 ...
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 ...
A 2-year-old boy died from a rare disease with flu-like symptoms, just weeks before his third birthday. Hudson Hughie Martin, ...
The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.
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.
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