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Addressing rare pediatric non-verbal spectrum disorder

What is a pediatric non-verbal disorder?Reading together

The signs and symptoms of a pediatric non-verbal learning disorder do not match those of any well-described condition and currently there is no formally accepted diagnosis. This is because there are difficulties in characterizing this condition that still challenge the medical community. Pediatric non-verbal disorder is accompanied with developmental delay, most notably where the toddlers never speak or will start speaking and then regress. The diagnosis is based on the identification of deficits in social perception, social judgment and social interaction skills, based on a toddler’s recognition of significant perception problems, faulty understanding of facial expressions, tone of voice and speaker’s intention. Low scores in specific motor performance tests involving both hands suggest bilateral brain involvement and confirm the presence of motor coordination impairments, with worse performance in the right hemisphere compared to the left. Children with non-verbal disorder are described as clumsy and uncoordinated. Visuospatial deficits are the major characteristic of children with non-verbal disorder, even in the absence of severe motor problems. Differences between verbal and performance (non-verbal) IQ scores are not a requisite for the diagnosis of non-verbal learning disorder; still this finding has been particularly emphasized in affected children.

What causes non-verbal disorder?Neuron New 2

Causes of non-verbal learning disorder have been linked to several complications that range from a specific mutated gene as with Fragile X Syndrome and Dravet Syndrome or autoimmunity, where the body’s immune system is attacking parts of the brain. Trauma, microbial infections and enviromental factors have also been linked to non-verbal learning disorder. Ongoing research is helping to further explain the root cause of why children become non-verbal or minimally verbal.

Who is most likely to get non-verbal disorder?

Children born into families where there is a genetic history of autism or epileptic spectrum disorders or that have a sibling that has been diagnosed with an autistic or epileptic spectrum disorder have a much higher chance of becoming non-verbal.

Prevalence

Among the >60,000 US children who develop Autism Spectrum Disorders (ASD) every year, 20,000 become non-verbal.

Current standard of care

Of the estimated 20,000 who become non- or minimally verbal, they will require assisted living for the rest of their life. The lifetime cost of that care is estimated at $10 million per person. Cognitive intervention is the only form for treatment that has shown to help improve speech capability and social interaction, however, it has not been able to alleviate the lifetime burden of $10 million per person for cost of care.

No therapies are currently available to treat this condition.

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The hard facts and emotional truth

Currently, there are no drugs available to treat this condition. As a result, lifetime costs of assisted living and supplemental healthcare average $10 million per person. This is compounded by an additional $10 million during the lifespan of the person due to loss in productivity. Not measuring the severe emotional strain of never talking to your child. This pediatric non-verbal disorder, where children lose or don’t develop speech and manifest with ASD symptoms is rare and limited to approximately 20,000 children a year in the US and about the same in Europe.

Method of action

QBM-001 is being studied with genetically identified children at high-risk of developing ASD during the toddler years. It is believed that QBM-001 will regulate faulty membrane channels that are known to cause migraines and/or seizures. QBM-001 acts as an allosteric regulator of these faulty channels in the brain to potentially alleviate the condition and allow toddlers to actively develop language and speech and avoid life-long speech and intellectual disability of being non-verbal. QBM-001 also is believed to reduce inflammation in the brain and by so doing may reduce the amount of long-term nerve loss.