Table 6.

Therapeutic strategies

S. No.	Disorder		Therapy	References
Recommended therapies
	ALD	Asymptomatic males	Lorenzo oil and dietary restriction of very long chain fatty acids	Moser et al., 2005; Sassa et al., 2014
		Males with adrenal insufficiency	Oral corticosteroid replacement	Raymond, Moser, & Fatemi, 1993
		Cerebral ALD	HSCT effectively arrests progression of demyelination if initiated early in course of disease	Shapiro et al., 2000; Peters et al., 2004; Miller et al., 2011
		Adrenomyeloneuropathy	Corticosteroid replacement for adrenocortical insufficiency Supportive treatment	Raymond, Moser, & Fatemi, 1993
2	MLD	Early onset forms	Cholecystectomy for preventing complications like gall bladder dysfunction, polyps and carcinoma Variable outcomes and several complications in patients treated with allogenic HSCT	Peters et al., 2004
		Juvenile or adult forms	Allogenic HSCT halts demyelination in minimally symptomatic Individuals	Peters et al., 2004; Weinberg 2005
3	Krabbe disease	Infantile onset	HSCT prevents progression of disease in presymptomatic individuals	Escolar et al., 2005
		Late onset	HSCT may be beneficial	Krivit et al 1999; Krivit 2004
4	Aicardi-Goutieres syndrome		Systemic corticosteroids are effective for treatment of autoimmune manifestations but not for neurologic symptoms	Chahwan and Chahwan 2012
5	Vanishing white matter disease		Preventive care to avoid trauma and infections Treatment of ovarian failure	van der Knaap et al., 2019
6	Canavan disease		Oral lithium citrate showed improvement in myelination in 6 patients but minimal clinically effectivity in tolerable lithium dosage	Assadi et al.,2010; Pleasure et al., 2020
7	Cerebrotendinous xanthomatosis		Oral chenodeoxycholic acid Cholic acid	Berginer et al 1984; Mandia et a., 2019
8	Adult onset polyglucosan body disease		Triheptanoin, a 7-carbon triglyceride has been found to be beneficial in slowing the clinical course	Roe et al 2010; (NCT00947960)
9	Hypomyelination with brain stem and spinal cord abnormalities and leg spasticity		Steroids may be beneficial in patients with subacute disease onset	Wolf et al., 2014
12	Pol-III related leukodystrophies		Supplemental hormonal therapies including growth hormone, thyroid hormone and hormone replacement therapy for hypogonadism	Bernard and Vanderver, 1993

X-ALD – X-linked adrenoleukodystrophy; HSCT- Hematopoietic stem cell transplantation; MLD- Metachromatic leukodystrophy