A renewal of the FDA’s pediatric rare disease voucher program is couched within a massive $1.2 trillion spending bill the ...
Jaguar has received the initial $16M payment related to the company's recently executed US out-license agreement for ...
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...
The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.
PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...
A 2-year-old boy died from a rare disease with flu-like symptoms, just weeks before his third birthday. Hudson Hughie Martin, ...
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 ...
Mendra, Inc. ("Mendra"), a biopharmaceutical company built to advance promising therapies for rare disease medicines, utilizing artificial intelligence (AI), today announced its launch in conjunction ...
Outbreak of a rare fungal disease in Tennessee leaves one dead and dozens sick - The infection can be particularly serious for people with compromised immune systems and can spread easily though inhal ...
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 ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results