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Clinical Neuroscience

MARCH 30, 2018

[Nusinersen in the treatment of spinal muscular atrophy]

SINKÓ Gabriella, KISS Zsuzsanna, BERNADETTE Kalman

[Until recently, the diagnosis of spinal muscular atrophy (SMA) has been associated with severe life-long motor disability in adults and with early death in infants. The new experimental therapeutic approaches of the last few years have become more and more promising, while nusinersen was approved for the treatment of SMA in December 2016 in the USA, and in May 2017 in Hungary. Our paper presents mechanisms and clinical benefits of this new medication, and highlights some of the other therapeutic strategies still in experimental stages.]

Lege Artis Medicinae

FEBRUARY 22, 2013

[New possibilities in treating hyperlipidemia]


[In the fight against atherosclerosis, statin therapy is one of the most important elements. On the basis of data from the past few years the clinical introduction of a more effective statin is not expected, however, in order to improve cardiovascular prevention further development of agents that reduce LDL-cholesterol levels more effectively than currently used statins is warranted. The need for the development of new cholesterol-lowering therapeutic options is also supported by the existence of statin intolerance. The currently available combination therapies do not provide additional mortality benefits compared with statin monotherapy. The new solutions include fourth-generation statin molecules that primarily aim to enhance the NO-donor capacity of statins, and to reduce their muscle toxicity. Certain compounds that affect cholesterol synthesis (squalene synthase inhibitors, MTP inhibitors, ACAT inhibitors) need to be further analysed because of the risk of side effects. The use of an antisense oligonucleotid that blocks the mRNA of apoB, the main protein on the LDL-particle and antibodies that inhibit the protein PCSK9 that promotes the intracellular breakdown of the LDL-receptor seems to be much more promising. Besides the lowering of LDLcholesterol level, studies have focused on the benefits of increasing HDL-cholesterol levels. Unfortunately, recently completed analyses show that new forms of the strong HDL-C increasing nicotinic acid have not provided any additional benefit when added to statin therapy. Similarly, the adverse effects associated with the promising CETP inhibitors and the lack of additional benefit when combined with statins question the significance of this drug class. The necessity for an absolute increase of HDL-cholesterol levels needs to be revised on the basis of new data, in other words, the exact role of the HDL particle in atherosclerosis needs to be further investigated.]

Lege Artis Medicinae

FEBRUARY 20, 2012

[Potentials and limitations of the application of RNA interference]

KISS-TÓTH Éva, JUHÁSZNÉ Szalai Adrienn, KOSKA Péter, KISS-TÓTH Emőke, BARKAI László, FODOR Bertalan

[The discovery of RNA interference revolutionised our knowledge about regulation. Small interfering RNA sequences, which have a central role in this phenomenon, go through a complex maturation process. By means of their antisense effect, they result in the degradation of homologuous RNA sequences or transcriptoinal repression in the cell. Due to its advantages, the application of gene silencing has rapidly become prevalent in basic research, especially in the field of molecular biology. Besides, an increasing effort has been made to utilise it in certain incurable diseases, viral infections, neurodegenrative disorders as well as in oncological diagnostics and therapy. However, in the interest of safe application, researchers in this area call our attention to the importance of answering a number of questions.]