N= You, Early Neurodevelopment Disorders

Question 1

What is the difference between diagnosis driven and person driven medicine medicine?

Answer 1

In diagnosis driven the patient is treated according to their formal diagnosis
In person driven medicine, the differences in a persons disease are respected and the signs and symptoms of a person are treated in accordance to them

Question 2

How and why is the concept of excitation and inhibition balance as target for PM in neurodevelopment disorder?

Answer 2

Excitatory (E) and inhibitory (I) systems are critical for regulating the flow of information in the brain. Without narrow control over the E/I ratio, runaway excitation or quiescence would occur, impeding adequate information processing.
In clinical terms, disruption of E/I balance has become a dominant theory on the pathogenesis of various neurodevelopmental disorders

Question 3

What is an EEG? What is measured?

Answer 3

a test that measures electrical activity in the brain using small, metal discs (electrodes) attached to the scalp

Question 4

What is the effect goal of modern trials?

Answer 4

the individual effect
(not the average effect)

Question 5

Autism

Answer 5

• not a disease - certainly not a single disease

DSM-5 (2013):
Autism Spectrum Disorder (ASD)
Poor social communication and interaction (all 3)
1. Clear deficiency in nonverbal and verbal social communication 2. Lack of social reciprocity
3. Poor development and maintenance of peer relationships

Limited repetitive behavior patterns, interests and activities (at least 2)
1. Stereotypical motor or verbal behavior or unusual sensory behavior 2. Excessive routines and ritualized behavior patterns
3. Limited, fixed interests
Clinically significant, persistent, present since early childhood

Question 6

What are the issues with a “diagnosis” in neurodevelopment disorders (eg. autism)?

Answer 6

• random cut-off in a continuum
• individuals with the same diagnosis have the same blocks but differently featured
• shell of associated diseases - cannot view disease in isolation

Question 7

What is missing from the autism diagnostic criteria?

Answer 7

• heterogeneity
• quantitive
• culture believes (what is odd in one culture common in another)
• severity

Question 8

What is traditional medicine?

Answer 8

• diagnosis driven
• standard therapy
• disease A = drug/therapy 1(Rx)

Question 9

Stratified Medicine

Answer 9

• Subtype driven
• dividing diagnosed people in subgroups - to target with different sub meds (Rx)
• eg. ‘hormone-receptor positive’, some are ‘HER-2 positive’, and some are neither

Question 10

Precision/ Personalised Medicine

Answer 10

• patient driven
• consideration of subtypes and individual qualities, environmental factors and lifestyle
• therapy tailed to each individual patient

Question 11

N = You

Answer 11

precision center for developmental disorders

Question 12

(multiple) N = 1 design

Answer 12

• patient is their own control
• clinical trial in which a single patient is the entire trial, a single case study. A trial in which random allocation can be used to determine the order in which an experimental and a control intervention are given to a patient is an N of 1 randomized controlled trial. The order of experimental and control interventions can also be fixed by the researcher
• individual subject experiences several treatment conditions, such as active treatment and placebo, in order to assess the individual’s specific response to each treatment
• N = You in progress: who responds to what and how
• assessment:
• Clinical effect
• Proms
• Conventional questionnaires
• EEGs
• Cognitive functioning