Researchers identify bone-forming cells as the driver of scoliosis caused by a genetic disorder, which can be prevented ...
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RAS-MAPK inhibition halts spinal deformity in neurofibromatosis type 1 mouse model
Spinal deformities such as scoliosis and kyphosis are among the most serious complications of neurofibromatosis type 1 (NF1), a genetic disorder that affects about one in 3,000 people.
Pasithea Therapeutics Corp. has announced the successful enrollment and initial dosing of three subjects in Cohort 6 of its Phase 1 clinical trial for PAS-004, a candidate for treating ...
When the NF1 gene was discovered in 1990, it was quickly learned that the gene product functions as a regulator of a protein called Ras. Ras is a key component of a cell signaling pathway that ...
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Cutting-edge treatment approach addresses neurobehavioral symptoms in neurofibromatosis type 1
A new study led by Indiana University School of Medicine researchers revealed a potential strategy to address neurobehavioral challenges associated with neurofibromatosis type 1, or NF1, a genetic ...
The US Food and Drug Administration has approved mirdametinib for the treatment of neurofibromatosis type 1 (NF1) in adults and children. The drug, developed by SpringWorks Therapeutics, is the first ...
Selumetinib's approval now includes children as young as 1 year old with NF1 and symptomatic, inoperable plexiform neurofibromas. The drug targets the RAS/MEK/ERK pathway, providing a non-surgical ...
Neurofibromatosis type 1 (NF1) is a genetic condition that causes tumors to grow along nerves in the skin, brain, and other parts of the body. These tumors are usually noncancerous (benign), but they ...
Neurofibromatosis type 1 (NF1) is a genetic condition that affects the skin, nervous system, and other parts of the body. The condition, formerly called Von Recklinghausen disease, causes changes in ...
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