… in the brain has become a high research priority to inform the … and epilepsy compared with
non-epilepsy Duchenne patients [… of the water channel aquaporin-4 and the Kir4.1 potassium …

… epilepsy precision medicine pipeline, from gene discovery to … muscular atrophy (SMA), 56
Duchenne muscular dystrophy, … priority areas, such as understanding the causes of epilepsy, …

… Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive
muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense …

… impact upon brain function. As cytokines can modify channels and receptors at both pre-
and post-synaptic membranes [159], this could facilitate deficits in cognition linked to a loss of …

… a pure seizure reduction by different AEDs to the discovery of … approved medication for
Duchenne muscular dystrophy, has … the rare pediatric disease priority review voucher by the FDA. …

… before the March 2022 priority date of this patent application. … and seizures, but the cause
and effect relationship is not clear. … the treatment of Duchenne muscular dystrophy and related …

… the clinical significance of genetic variants discovered through … for more efficacious treatments
for DRE remains a priority. … spinal muscular atrophy, Duchenne muscular dystrophy and …

… specific therapies is not a priority for pharmaceutical companies [… nervous system may lead
to seizures, and the diagnosis is … Duchenne muscular dystrophy (DMD) is a dystrophinopathy …

… Total body water in SMA type I and SMA type II patients is … muscular atrophy with progressive
myoclonic epilepsy (SMA-… be a priority to better medically manage them going forward. …

… Examples are Duchenne muscular dystrophy and … epilepsy with febrile seizures plus (GEFS+),
seizures may persist beyond early childhood. Mutations of at least four ion channel …