Ovid Therapeutics has recently announced that it will host a webinar in an effort to educate people about Angelman syndrome and their possible treatment for it, OV101. Speakers will focus on topics like treatment practice, the biological mechanisms, the role of tonic inhibition, measurement scales, and updates about the development of OV101. The webinar will take place on October 22 at 1 PM and can be found here.
OV101 is a delta-selective GABAA receptor agonist that is currently being developed for the treatment of Angelman syndrome and Fragile X syndrome. It was created to regulate tonic inhibition, a process that plays a role in a number of neurodevelopmental disorders.
Ovid’s treatment is the first and only medication that is intended to disrupt tonic inhibition, making its development very exciting to many. Positive results from prior trials have furthered this excitement, and researchers are now awaiting data from the Phase 3 NEPTUNE trial.
This treatment has also been granted the Rare Pediatric Disease designation for Angelman syndrome, and the Orphan Drug designation for both Angelman and Fragile X syndrome. In Europe, the EC has granted OV101 the Orphan Drug designation for Angelman syndrome as well.
About Angelman Syndrome
Angelman syndrome (AS) is a neuro-genetic disorder. Due to its rarity, it is often misdiagnosed as cerebral palsy or autism. It is characterized by seizures, developmental disabilities, and neurological problems and occurs when a gene on chromosome 15 is either defective or missing. The reason for this change in chromosome 15 is unknown, as there does not seem to be a hereditary component.
The first signs of AS present in early childhood, as children do not crawl or talk at the normal age. Seizures will come later in childhood. Children with this syndrome are often very happy and like to laugh and smile. Other symptoms of this condition include minimal or no speech, small head size, difficulty with walking and moving, tongue thrusting, hand flapping and uplifted arms when walking, stiff and jerky movements, sleep disorders, and feeding difficulties.
After a doctor notices the characteristic symptoms of AS, they will order genetic tests to confirm a diagnosis. These tests may be a DNA methylation test, gene sequencing tests, and fluorescence in situ hybridization (FISH) tests. After a diagnosis is confirmed, treatment is meant to manage the symptoms of the syndrome. Common treatments include anti-seizure medications and behavioral, physical, and communication therapy.
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