These patients were only considered for gene therapy after there was no HLA match for a traditional transplant. In two reported cases, the gene therapy was successful, with a resolution of the demyelination process up to two years after the procedure. Although the gene therapy was successful in resolving the neurological symptoms, plasma VLCFA levels remained elevated.Elivaldogene autotemcel is pending authorization by the European Commission as of May 2021. Adrenal insufficiency
Treatment of the adrenal insufficiency that can accompany any of the common male phenotypes of ALD does not resolve any of the neurological symptoms. Hormone replacement is standard for ALD patients demonstrating adrenal insufficiency. Adrenal insufficiency does not resolve with successful transplant; most patients still require hormone replacement. Epidemiology
ALD has not been shown to have an increased incidence in any specific country or ethnic group. In the United States, the incidence of affected males is estimated at 1:21,000. Overall incidence of hemizygous males and carrier females is estimated at 1:16,800. The reported incidence in France is estimated at 1:22,000. Asymptomology
There are documented asymptomatic males who present no ALD symptoms well into their 60s and 70s. Its not understood how they can have an ABCD1 gene variant and possess elevated VLCFAs and not exhibit either Cerebral ALD, Adrenal Insufficiency, or Adrenomyeloneuropathy symptoms. Daughters of asymptomatic males become obligate carriers, who may themselves be asymptomatic and who can pass the variant onto their children, which then silently perpetuates ALD. Sons of asymptomatic males only receive their fathers Y chromosome and therefore cant inherit ALD.
This drug is also used off-label in cats and dogs, both by injection and orally. The cross-species nature of IFN-α allow it to work in non-human animals, but the period of usefulness is limited by the production of antibodies against this foreign protein. See also
Interferon
Ropeginterferon alfa-2b

References
External links

Intron-A Summary of Product Characteristics
Nagata S, Taira H, Hall A,  et al. (Mar 1980). "Synthesis in E. coli of a polypeptide with human leukocyte interferon activity". Nature. 284 (5754): 316–20. Bibcode:1980Natur.284..316N. doi:10.1038/284316a0. PMID 6987533. S2CID 4310807.