RESILIENT a new clinical trial researching an investigational treatment option for Spinal Muscular Atrophy (SMA). For children and adults, age 4-21.
Areas covered: This article covers the publically available data on therapeutic strategies that address the underlying cause of SMA and clinical data available on approved treatments and drugs in the pipeline. Expert opinion: The newer therapeutic options in SMA have a good safety profile and deliver...
Over the last few years, several new treatments for SMA have dramatically improved the prognosis of affected patients3. Nusinersen4was the first drug to be approved by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in December 2016 and June 2017, ...
Because it is structurally different from the other treatments, it could potentially be more effective, and possibly have uses in indications other than epilepsy that also involve the Kv7 ion pathway. Vlad Coric, M.D., CEO and chairman of the board at Biohaven, said, “With this transact...
Influenza has a long history of causing morbidity and mortality in the human population through routine seasonal spread and global pandemics. The high mutation rate of the RNA genome of the influenza virus, combined with assortment of its multiple genomi
For instance, DLAT expression is notably elevated in gastric cancer, suggesting its potential as a therapeutic target. Furthermore, recent studies have shown that DLAT expression is high in patients who exhibit resistance to anti-PD-L1/PD-1 treatments, implying that DLAT could serve as a ...
This work with a mouse model of SMA by Lutz and her colleagues lay the foundation for the successful clinical trials of Spinraza. “A colleague once asked me why I was working so hard on a rare disease like SMA to benefit so few patients,” Lutz recounts. “Th...
Chan B, Kalabalikis P, Klein N, Heyderman R, Levin M. Assessment of the effect of candidate anti-inflammatory treatments on the interaction between meningococci and inflammatory cells in vitro in a whole blood model. Biotherapy 1996;9(4):221–228. ...
treatments. In addition, it was demonstrated by Wang et al. that somatic SF mutations increased acute myeloid leukemia (AML) cells sensitivity to splicing inhibitors, suggesting these mutations as biomarkers for sensitivity to Indisulam and other sulfonamides [179]. Anke Nijhuis et al. demonstrated ...
New and prospective future treatments New and future neuraminidase inhibitors Laninamivir Recently, a new NA inhibitor, laninamivir (Inavir; Daiichi-Sankyo and Biota; Figure2), has been approved for use in Japan [50,51], and is currently being developed in the US. It is a highly promising an...