NEW YORK, NY — Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the United States Food and Drug Administration (FDA) has granted Investigational New Drug (IND) clearance to directly proceed for a registrational clinical trial evaluating Ryoncil® (remestemcel-L-rknd) in Duchenne muscular dystrophy (DMD),...
treatment News
NEW YORK, NY — Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has received seven years of orphan-drug exclusive approval from the FDA for Ryoncil® (remestemcel-L) for treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age...
Hefei, China – A research group led by Prof. WANG Hui from the Hefei Institutes of Physical Science (HFIPS) of the Chinese Academy of Sciences has introduced a metal-free nanozyme based on graphene quantum dots (GQDs) for highly efficient tumor chemodynamic therapy (CDT). The study was published in Matter. GQDs...
Zurich, Switzerland – MetrioPharm AG, a pharmaceutical company developing drugs for inflammatory and infectious diseases, announced today that the European Medicines Agency has granted Orphan Drug Designation (ODD) to its lead compound, MP1032 for the treatment of children affected by Duchenne muscular dystrophy (DMD). This milestone follows the U.S. FDA’s...
Chongqing, China – Metronomic chemotherapy (MC) is emerging as a groundbreaking treatment for pediatric neuroblastoma, offering a more accessible and manageable option for children with high-risk and relapsed/refractory forms of the disease. This innovative approach could revolutionize care, particularly in low-resource settings where traditional treatments are often out of reach....
FORT LEE, N.J. — Mezzion Pharmaceuticals is a rare disease company conducting the confirmatory pivotal phase 3 clinical trial in Fontan subjects: The Fontan Udenafil Exercise Longitudinal Assessment Trial- 2, or FUEL-2 trial. The company is pleased to announce Dr. Rahul Rathod, a pediatric cardiology specialist and leader with over...
MIAMI, FL – Researchers from Miami Cancer Institute, part of Baptist Health South Florida, today published a study in Trends in Cancer that analyzes the use of tissue-agnostic therapeutics in patients with primary brain tumors (PBTs). The publication describes the current and potential impact of tissue-agnostic therapies on the management...
Last week, the Michigan House passed (88-20) House Bill 4167, which would create a Rare Disease Advisory Council (RDAC) within Michigan’s Department of Health and Human Services (MDHHS). The council would be required to conduct research, identify priorities, and make policy recommendations regarding access, treatments, and services for rare diseases...
EAST LANSING, Mich. – A team of Michigan State University scientists has unveiled a potential game-changer in the fight against glioblastoma, the most common and currently incurable form of brain cancer. A team of Michigan State University scientists has unveiled a potential game-changer in the fight against glioblastoma, the most...
Bozeman, Mont. and Vancuver, BC — Microbion Corporation announced that the US Patent and Trademark Office (USPTO) issued United States Patent No. 11,464,749 to Microbion with claims to the use of Microbion’s proprietary pravibismane inhaled composition for treatment of pulmonary infections. The patent, entitled “Bismuth-thiol compositions and methods of use,”...