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Rare Genomics Institute Helps in Hailey-Hailey Disease Treatment Study

Publication date: 13 Dec 2017

Rare Genomics (RG) was approached by the Hailey-Hailey Disease Worldwide Support Group to help conduct a study to test low-dose naltrexone (LDN).  Hailey-Hailey disease (HHD) is a rare genetic skin disorder characterized by blisters or erosions on the skin. These skin abnormalities can come and go throughout a person’s life. Certain triggers such as heat or friction can worsen the problem. 

RG did some research on the biological and clinical rationale for LDN as a treatment for HHD, and determined there was a reasonable basis to conduct a study. However, doctors and clinical investigators have been reluctant to conduct a study since there are no publications. RG decided to publish a review article summarizing the rationale for LDN in HHD. This article was accepted by the medical journal, JAMA Dermatology. 

HHD is caused by a change in a gene called ATP2C1. This altered gene usually occurs spontaneously.  Because the gene is altered, the protein produced by the gene is defective or produced in insufficient levels. This can be called reduced ATP2C1 gene activity. Despite the studies on ATP2C1 and its relation to HHD, few treatment options exist. Without an established treatment model, physicians rotate through numerous therapies including corticosteroids, all while the patient struggles with the debilitating symptoms and deteriorating quality of life. 

RG had received testimonial evidence in which patients with HHD taking LDN reported up to 90% healed skin and a reduction in body weight, depression, and suicidal thoughts. Despite these promising results, there were no published case studies or clinical studies studying LDN to treat HHD. Such studies are necessary to better understand the mechanism of action and effects of LDN in HHD, and to prove the long-term safety and effectiveness of a treatment. Many physicians have hesitated to prescribe LDN because of the lack of published studies. 

Following the publication from RG describing LDN as a treatment for HHD, additional articles have been published that detail several case studies reporting the success of LDN to treat HHD. Hopefully, these anecdotal reports will lead to clinical studies in LDN as a potential treatment for individuals who have HHD. 


Ibrahim O, Hogan SR, Vij A. Low-dose naltrexone treatment of familial benign pemphigus (Haile-Hailey Disease). JAMA Dermatol. 2017;153(10):1015-1017. doi:10.1001/jamadermatol.2017.2445.

Albers LN, Arbiser JL, Feldman RJ. Treatment of Hailey-Hailey Disease with low-dose naltrexone. JAMA Dermatol. 2017;153(10):1018-1020. doi:10.1001/jamadermatol.2017.2446.

Campbell V, McGrath C, Corry A. Low dose naltrexone: a novel treatment for Hailey-Hailey disease. Br J Dermatol. 2017. doi: 10.1111/bjd.16045.