All people have two copies of every gene – one copy inherited from each parent. The NF1 gene mutation is dominant, which means that only one of the two copies of the gene needs to have the mutation to ...
UAB genetic scientists Bob Kesterson, Ph.D., and Deeann Wallis, Ph.D., have developed animal models with patient-specific NF1 mutations for understanding both the structure and function of the NF1 ...
Inactivating germ line mutations in the neurofibromatosis type 1 (NF1) gene are responsible for the many manifestations of this disease that range from intellectual deficits, to autism, to bone ...
Gomekli is the second ever FDA-approved treatment for rare tumor disease, NF1-PN and the first to be approved for both adult ...
The Food and Drug Administration (FDA) approved a new drug to treat a disorder causing the growth of noncancerous tumors on ...
Using advanced machine-learning techniques, the team will analyse the brain structure of over 10,000 MRI scans ... for researchers to deepen our understanding of how changes in the NF1 gene impact ...
Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder neurofibromatosis type 1 (NF1). More specifically, Gomekli is for those age 2 ...
Neurofibromatosis Type 1 (NF1) is a genetic disorder that affects approximately 1 in 3,000 individuals worldwide. It is characterized by the development of benign and malignant tumors, skin ...
Using advanced machine-learning techniques, the team will analyse the brain structure of over 10,000 ... our understanding of how changes in the NF1 gene impact brain development.