May 16, 2022
Rocket Reports Updates of Data on Multiple Gene Therapy Programs
Read More
May 13, 2022
FDA Approves Oral Form of Radicava for Treatment of Adults with ALS
Read More
May 12, 2022
Caribou Reports Positive Initial Data for Gene-Edited Allogeneic CAR-T Cell Therapy
Read More
May 12, 2022
Takeda Notifies Ovid of Delays in Pivotal Study of Therapy for Rare Epilepsies
Read More
May 11, 2022
Takeda Halts Development of Hunter Syndrome Therapy
Read More
May 11, 2022
FDA Extends PDUFA Date for Amicus’ Experimental Therapy to Treat Pompe Disease
Read More
May 10, 2022
UCB Reports Positive Results in Phase 3 Generalized Myasthenia Gravis Study
Read More
May 10, 2022
FDA Approves BMS Drug for Rare Genetic Heart Condition
Read More
May 9, 2022
Mereo Reports Positive Data from Phase 2 Study of Alvelestat in AATD-related Emphysema
Read More
May 9, 2022
FDA Lifts Clinical Hold on LogicBio’s Trial in Pediatric Patients with Methylmalonic Acidemia
Read More
May 5, 2022
Ultomiris Met Primary Endpoint in Phase 3 Trial in Adults with Rare Autoimmune Condition NMOSD<br><br><em>Rare Daily Staff</em>
Read More
May 3, 2022
FDA Grants DepYmed Rare Pediatric Disease Designation for Rett Syndrome Candidate
Read More
Load More
X