Mutations in the NF1 gene lead to neurofibromatosis type 1, which often leads to bone abnormalities and spine deformity. Efforts to treat or even prevent the disease have been stymied by lack of understanding about how the disease occurs and progresses.
GSK plc’s Blenrep (belantamab mafodotin) is heading back to the market three years after being withdrawn, with the EMA’s Committee for Medicinal Products for Human Use recommending approval of the antibody-drug conjugate in combination therapy for the treatment of adults with relapsed or refractory multiple myeloma.
The FDA has awarded orphan drug designation to Healx Ltd.’s HLX-1502 for the treatment of neurofibromatosis type 1 (NF1). HLX-1502, discovered through the company’s proprietary artificial intelligence (AI) drug discovery platform, has a first-in-class mechanism and supporting data that indicate the potential for a good safety profile.
In a study published in the online edition of Proceedings of the National Academy of Sciences on Jan. 23, 2023, a team of scientists from Frederick National Laboratory for Cancer Research and the University of California, San Francisco (UCSF) described how neurofibromin 1 (NF1) missense mutations act in a dominant negative manner through dimerization with wild-type neurofibromin.
Researchers from Qualigen Therapeutics Inc. and University of Louisville presented the discovery and preclinical evaluation of a novel pan-RAS inhibitor...
Toosonix A/S has launched two trials as it moves to gather data enabling its high-intensity focused ultrasound (HIFU) device to get approval for use in clinical dermatology indications. The company reported on April 19 that the first patients have been treated in a trial of its System One-M device in treating basal cell carcinoma (BCC). Meanwhile, the first patient has been cleared for treatment on April 28 in a study in neurofibromatosis, an inherited condition that causes benign tumors to form on the skin.
The U.S. National Institutes of Health had its hands full with the Rapid Acceleration of Diagnostics (RADx) program as the COVID-19 pandemic unwound, but the agency’s other work on diagnostics is bearing fruit. NIH said Aug. 31 that its collaboration with an academic research institute has led to development of a test that predicts which patients suffering from neurofibromatosis will develop cancers with metastatic potential.
Following a priority review, partners Astrazeneca plc and Merck & Co. Inc. have gained a green light from the FDA for U.S. marketing of the oral MEK1/2 inhibitor Koselugo (selumetinib), the first FDA-approved treatment for the rare genetic disorder neurofibromatosis type 1 (NF1).
