MGH Team Defines the Mechanisms of Action of Key Genetic Abnormality in Ewing Sarcoma
A Massachusetts General Hospital (MGH) research team has used epigenome editing tools to investigate how the genetic abnormality that drives Ewing sarcoma – the second most common bone cancer in children and young adults – unleashes tumor growth. In their paper in the journal Genes & Development the researchers show that blocking the fusion protein…
Scientists Discover Potential Therapy for Human Copper Metabolism Disorders
New Hope For Rare Disorder
Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent painful swellings of the skin and mucous membranes. Without treatment, patients’ quality of life is noticeably compromised: Angioedema may not only be disfiguring; in the gastrointestinal tract it may lead to severe abdominal colic ad in the upper airways it can even be fatal…
FDA Approves First Targeted Treatment for Patients with Acute Myeloid Leukemia
Enzyme Identified as Possible Novel Drug Target for Sickle Cell Disease
FDA Approves First Drug Comprised Of Active Ingredient Derived from Marijuana
The U.S. Food and Drug Administration today approved Epidiolex (cannabidiol) [CBD] oral solution for the treatment of seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome, in patients two years of age and older. This is the first FDA-approved drug that contains a purified drug substance derived from marijuana.…