During this study, treatment with satralizumab reduced the number of NMOSD relapses by 74% in participants who were anti-AQP4 positive compared to treatment with a placebo (inactive treatment).A study of satralizumab as an adjuvant to immunosuppressant treatment for NMOSD included 76 adult participants; 52 were anti-AQP4 positive. Immunosuppressant treatment in combination with satralizumab reduced the rate of relapses in participants who were anti-AQP4 positive by four-fifths compared to immunosuppressant treatment alone.The U.S. Food and Drug Administration (FDA) approved satralizumab based on evidence from two clinical trials (Trial 1/ NCT02073279 and Trial 2/NCT02028884) of 116 participants with NMOSD who were anti-aquaporin-4 (AQP4) antibody positive. The trials were conducted at 62 sites in the United States, Canada, Europe and Asia. Participants received at random either satralizumab or placebo injections according to the schedule. Neither the participants nor the healthcare providers knew which treatment was being given. In the second trial, all participants were also receiving their current immunosuppressive medications for the treatment of NMOSD. The benefit of satralizumab was evaluated by measuring the time to the first attack and comparing it to placebo.There was no evidence of a benefit in participants who were anti-AQP4 antibody negative in either trial. Society and culture
Legal status
Satralizumab was approved for the treatment of AQP4-IgG-seropositive NMOSD in Canada, Japan, and Switzerland.Satralizumab was approved for medical use in the United States in August 2020. The U.S. Food and Drug Administration (FDA) granted the application for satralizumab fast track and orphan drug designations.
loss-of-function mutations in one of the two normally paired TSC1 or one of the two normally paired TSC2 tumor suppressor genes. As a part of their functions, the TSC1 and TSC2 tumor suppressor genes act to suppress the abnormal growth of cells by contributing to the suppression of the mammalian target of rapamycin (i.e. mTOR) protein that promotes cell growth and proliferation. Inactivation of one of the TSC1 or TSC2 genes appears responsible for unleashing mTor and thereby promoting the growth of the many strictly benign lesions, including KTs, that develop in individuals with tuberous sclerosis. mTOR is inhibited by rapamycin, a drug which has been used as a topical application to successfully treat a few cases of Koenens tumors.A small number of cases in individuals including those that do not have tuberous sclerosis may develop KTs as reactions to local traumas. Treatment
The treatment of KTs has varied depending on their size, numbers, locations, symptoms, damage to tissues, and disfiguring effects. Excision may be the treatment of choice for surgically accessible lesions. However, following surgical removal, KTs have a high rate of local recurrence, particularly in cases where the lesions are not completely removed. Other treatment methods, which may be used in combination with surgical removal and/or with each other include: carbon dioxide-based laser vaporization; electrocauterization; shave excision of the tumor with phenolization (i.e.