[7] Her work helped elucidate the genes and genetic mechanisms responsible for a number of devastating neurological disorders, such as Rett syndrome and spinocerebellar ataxia type 1.

[8] Zoghbi's discoveries have provided new ways of thinking about other neurological disorders such as Parkinson's disease, Alzheimer's, autism and intellectual disability, which could lead to new therapeutics and better, more efficient treatments.

[11] Her mother convinced her to study biology instead, on the grounds that 'a woman growing up in the Middle East should pick a career ensuring independence and security, while she can always write on the side'.

Although she and her classmates decided to stay at the university, after her brother was injured by shrapnel, their parents sent them to live with their sister in Austin, Texas, with plans to return the following summer.

However, in October, it was confirmed that she was still unable to return to Lebanon due to the war, and US medical schools had begun their fall term 2 months earlier.

[13] Despite her continued desire to return to Lebanon the next summer, on the advice of professors at AUB, she stayed at Meharry and earned an MD degree in 1979, after which she joined the Texas Children's Hospital at the Baylor College of Medicine as a pediatric resident.

[3] An article she published in 1985[15] attracted many Rett syndrome patients to Texas Children's Hospital, giving her access to a large number of cases.

[19] Further work by Zoghbi, Orr and their teams demonstrated that the misfolding, aggregation, and proteasomal degradation of the protein product of this gene, Ataxin 1, played a role in the disorder.

As Baylor's Hugo J. Bellen described the role of the atonal gene in balance in fruit flies, Zoghbi chose to study its mammalian homolog.

[21] Her team went on to find that, in addition to its involvement in balance and coordination, Math1 is also crucial to hearing,[22] the formation of secretory cells in the gut.,[23] and neonatal respiratory rhythm and chemosensitivity in the adult brain by regulating the development of a group of hindbrain neurons.

[28] In 2009, she found mice deficient of the Mecp2 gene (the mouse homolog of human MECP2) had lower levels of norepinephrine, dopamine and serotonin,[29] consistent with her clinical observations of patients of Rett syndrome in 1985.

[32] The subsequent study found that loss of ataxin-1 elevates BACE1 expression and Aβ pathology in mouse models, rendering it a potential contributor to risk and pathogenesis of Alzheimer's disease.