December 5, 2019
Regeneron Reports Positive Data with Pozelimab in Patients with a Rare Blood Disorder
Read More
December 4, 2019
Censa Reports Positive Results in Phase 2 Trial in Patients with PKU
Read More
December 2, 2019
FDA Accepts Incyte’s Cholangiocarcinoma Candidate for Priority Review
Read More
November 29, 2019
How a Brother’s Love Is Forging a Path for Customized Therapies for Rare Disease Patients
Read More
November 27, 2019
La Jolla Pharmaceuticals Halts Development of Beta Thalassemia Drug as CEO Departs
Read More
November 27, 2019
ChemoCentryx Reports Positive Data from Pivotal Trial of Avacopan in ANCA-Associated Vasculitis
Read More
November 26, 2019
FDA Approves Novel Treatment to Target Abnormality in Sickle Cell Disease
Read More
November 25, 2019
Alexion’s Soliris Wins Japanese Approval to Treat NMOSD
Read More
November 25, 2019
FDA Grants PTC Priority Review for SMA Therapy
Read More
November 21, 2019
Healx Launches Rare Treatment Accelerator
Read More
November 21, 2019
FDA Grants Newron Rare Pediatric Disease Designation for Sarizotan in Rett Syndrome
Read More
Load More
X