Antisense

Amyotrophic lateral sclerosis ( ALS ) is a rapidly progressing neurodegenerative disease that is characterized by motor neuron loss and...


Interim topline data from a randomized, double-blind, multicenter, exploratory phase 1b study evaluating the effects of oral Mongersen ( GED-0301...


Duchenne muscular dystrophy is caused by dystrophin deficiency and muscle deterioration and preferentially affects boys. Antisense-oligonucleotide-induced exon skipping allows synthesis...


Phase IIa clinical trial data on ATL1102 were published in the medical journal Neurology. The article highlights the successful outcomes...


Crohn’s disease-related inflammation is characterized by defective activity of the immunosuppressive cytokine transforming growth factor ( TGF )-beta1, due to...


On 13 December 2012, the Committee for Medicinal Products for Human Use ( CHMP ) has adopted a negative opinion,...


The stability and propagation of hepatitis C virus ( HCV ) is dependent on a functional interaction between the HCV...


Dravet syndrome is a severe epilepsy syndrome characterized by infantile onset of therapy-resistant, fever-sensitive seizures followed by cognitive decline. Mutations...


A new type of treatment significantly reduces the severity of muscle weakness in myasthenia gravis, giving hope for a new...


Researchers have developed a successful strategy for interfering with Ebola virus infection that protected 75 percent of nonhuman primates exposed...


A study has examined the function of a protein called HBZ, which is made by the human T cell leukemia...