A renewal of the FDA’s pediatric rare disease voucher program is couched within a massive $1.2 trillion spending bill the ...
PPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is ...
The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...
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 ...
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 ...
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 ...
Danish dermatology specialist Leo Pharma and its CEO Christophe Bourdon came to San Francisco to attend the J.P. Morgan ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results