Lonafarnib, sold under the brand name Zokinvy, is a medication used to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in people one year of age and older.The most common side effects included nausea vomiting, headache, diarrhea, infection, decreased appetite and fatigue.Lonafarnib was approved for medical use in the United States in November 2020, and in the European Union in July 2022. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses
Lonafarnib is indicated to be used to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain other processing-deficient progeroid laminopathies in people one year of age and older. Contraindications
Lonafarnib is contraindicated for co-administration with strong or moderate CYP3A inhibitors and inducers, as well as midazolam and certain cholesterol-lowering medications. History
Lonafarnib, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein. The effectiveness of lonafarnib for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in 62 patients from two single-arm trials (Trial 1/NCT00425607 and Trial 2/NCT00916747) that were compared to matched, untreated patients from a separate natural history study. Compared to untreated patients, the lifespan of Hutchinson-Gilford progeria syndrome patients treated with lonafarnib increased by an average of three months through the first three years of treatment and by an average of 2.5 years through the maximum follow-up time of 11 years.
Epilepsia partialis continua is a rare type of brain disorder in which a patient experiences recurrent motor epileptic seizures that are focal (hands and face), and recur every few seconds or minutes for extended periods (days to years). It is sometimes called Kozhevnikovs epilepsia named after Russian psychiatrist Aleksei Yakovlevich Kozhevnikov who first described this type of epilepsy. Signs and symptoms
During these seizures, there is repetitive focal myoclonus or Jacksonian march. After a seizure has subsided, Todds phenomenon may be observed, which includes transient unilateral weakness. Causes
There are numerous causes for this kind of seizure and they differ depending somewhat on the age at which the seizures begin. Epilepsy most often occurs at the extremes of life – in childhood or in very old age – but can develop at any time throughout ones life. Although these seizures are usually due to large, acute brain lesions resulting from strokes in adults and focal cortical inflammatory processes in children (Rasmussens encephalitis), possibly caused by chronic viral infections, edema, or autoimmune processes. They are very medication and therapy-resistant, and the primary therapeutic goal is to stop secondary generalization. There are also many other reasons why these seizures occur. For example, they could be due to genetics, infections, or problems with brain development. Commonly the cause is unknown. Genetic background determines such features as height, eye color, and potential to develop certain diseases like diabetes, but it also determines all the chemicals and structures that make up the brain, therefore playing a role in epilepsia partialis continua.