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Table 3 Details of empirical medications, categorized by class and by efficacy in Peak phase

From: Parental perspectives of episodic irritability in an ultra-rare genetic disorder associated with NACC1

Medication class

Number of patients

Single most effective medicine for each patient during peak of episodes

Additional effective medicationsa endorsed by parents

Non-opioid painkillers

5

Paracetamol (2)

 

Opioid painkillers

5

 

Hydromorphone

Oxycodone

Psychotropic medications and sedatives: anti-anxiety, antidepressant, anti-psychotic

10

Clonazepam (2)

Lorazepam

Diazepam (2)

Chloral hydrate

Nortriptyline

Lorazepam (3)

Diazepam

Chloral hydrate

Clonazepam

GERD/GI medications

9

Daily enema

 

Anti-epileptic, neuralgic, dystonia

11

Medical marijuana/THC (2)

Medical marijuana/THC (2)

Phenobarbital

Anti-adrenergic

7

Clonidine (2)

Clonidine (4)

Tizanidine

Beta blocker

2

Propranolol

Atenolol (tachycardia)

Miscellaneous

4

 

Atenolol (tachycardia)

Ondansetron injection (severe nausea)

Tetrabanazine

Non-prescription sleep aids

4

 

Dream (melatonin, gabapentin, chamomile extract)

  1. aThe medications are not mutually exclusive, and many patients were on multiple medications