Smile for GNAO1

What is GNAO1?

GNAO1 is a rare genetic neurodevelopmental disorder arising from a mutation of the eponymous gene.

GNAO1 disorder was first characterised in 2013.

Today roughly 200 people worldwide have been diagnosed with the GNAO1 disorder

There is no cure or treatment for GNAO1.

About us

Famiglie GNAO1 Association was established in 2019 by a group of families in Italy to better support other families in Italy afflicted by the GNAO1 disorder. Whilst focused on serving the patient community within Italy, we aim to collaborate with patient family groups, physicians and researchers in other countries to facilitate information sharing and ultimately to find a cure for this rare disease.

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Rare disease

In Europe a disease is classified as rare if it affects fewer than 1 in 2000 people. These diseases are rare but numerous, estimated to affect 3%-5% of the global population. There are currently over 7000 known rare diseases, and new ones are described in scientific publications every year. 80% of rare diseases are of genetic origin and emerge during childhood. It is estimated that in Italy there are 2 million rare disease patients and 30 million in Europe.

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In recent years, an increasing number of research groups and clinicians have focused their attention on this pathology, to try to shed light both on the neurological mechanisms that underlie it, and on its manifestation and evolution. This section collects works from the international scientific community, which is certainly an important resource for anyone who can deepen their knowledge of the disease.

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GNAO1 European Conference 2023

Famiglie GNAO1, the Italian GNAO1 patients association, is happy to announce the third edition of the GNAO1 European Conference, in Rome, on the 16th and 17th of June 2023 . Two days entirely dedicated to the ultra-rare disease caused by the GNAO1 gene mutation. An event open to clinicians, researchers, rehabilitation experts, families and everyone interested in this genetic disease.

The conference is structured as follows:

• The first day will focus on clinical aspects of the disease as well as on the latest updates on the ongoing research projects. Note that all the talks will be in English with real time translation to Italian and Spanish.
• The second day will cover rehabilitation topics and will go through what has been discussed the day before with an easier language, at the advantage of the non-technical audience. A session dedicated to accessible videogaming will complete the day. Note that the talks will be mostly in Italian with real time translation to English and Spanish.

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Events and news

28 February 2024

Famiglie GNAO1 Research Grant

Introduction Famiglie GNAO1 APS is a non-profit association founded in 2019 by a few families of kids affected by neurodevelopmental disorders related to the GNAO1 gene. The main aims of the association are: create a network among families and promote inclusion spread the awareness about this disease create a network among clinicians and researchers support […]

4 October 2020

ENG – Run 4 GNAO1 Warriors

On GNAO1 Awareness Day, Famiglie GNAO1 invites all its supporters to participate in the RUN 4 GNAO1 WARRIORS awareness initiative. A virtual run / walk, to support research on disease and help all our little, great warriors.

1 October 2020

ENG – GNAO1 EU Conference 2020

Famiglie GNAO1 hosted the second GNAO1 European Conference online from 1-3 October 2020. The principal objectives of this conference were: facilitate cross-pollination of research ideas and developments across the globe, continue to build a centre of innovative GNAO1 research and practice in the European Union and generate awareness, so that GNAO1 patients are able to […]

ENG – GNAO1: partono i primi progetti di ricerca sostenuti dall’associazione italiana

In soli due anni, la onlus “Famiglie GNAO1” è diventata un punto di riferimento per i pazienti e la comunità scientifica. La malattia correlata a GNAO1 è una patologia genetica molto rara che colpisce il neurosviluppo e con esordio in età pediatrica. Ad oggi, sono noti poco più di 200 pazienti al mondo affetti da questa malattia, i […]

Events and news


Every day the little GNAO1 patients, together with their families and with the support of therapists, doctors and educators, live the difficulties of their daily life with a warrior commitment but at the same time with the spontaneity and the smile of their being, first of all, children.

October 1st is the right day to help the association make the impossible possible!

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This map shows the location of the GNAO1 families currently in contact with Famiglie GNAO1. If you are a family with a recent GNAO1 mutation diagnosis, know that you are not alone. The families in this group are ready to give support and to share experiences and information. If you want to get in touch with us, you may do so via the contact form below.

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