Stanford, Calif. – An “anti-hunger” molecule produced after vigorous exercise is responsible for the moderate weight loss caused by the diabetes medication metformin, according to a new study in mice and humans. The molecule, lac-phe, was discovered by Stanford Medicine researchers in 2022. The finding, made jointly by researchers at...
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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...
DETROIT – Ryder Washington started kindergarten this year. The 5-year-old loves to dance, practice martial arts and is obsessed with the Statue of Liberty. A few months ago he was diagnosed with a rare disease which in most cases leads to leukemia. The child’s family is hoping someone will be able...
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...
MIAMI, FL – February 15, 2024 – A study co-led by researchers at Miami Cancer Institute, part of Baptist Health South Florida, found that ablative stereotactic magnetic resonance (MR)-guided adaptive radiation therapy may improve local control (LC) and overall survival (OS) in patients with borderline resectable (BRPC) and locally advanced...
NEW YORK — Scientists at Weill Cornell Medical College have developed a new mouse model of Parkinson’s disease (PD) that successfully reproduces the impairments of movement and the degenerative brain changes that occur in the human disease. Their research, performed in collaboration with investigators at Columbia University Medical Center, appears...
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...