Appoints Joseph Horrigan, MD to Board of Directors Appoints Joseph Horrigan, MD to Board of Directors

Categories: Blog, News Appoints Joseph Horrigan, MD to Board of Directors

CINCINNATI, OH – May 3 – is pleased to announce that Joseph P. Horrigan, MD has joined its Board of Directors. Dr. Horrigan’s appointment follows the excitement of several research and clinical trial advancements supported or developed through

Dr. Horrigan is a pediatric neuropsychiatrist with over thirty years’ of experience working with children and adults with neurodevelopmental and pervasive development delays. He also has twenty years of experience in the pharmaceutical and biotech industry. Dr. Horrigan formerly served as the Head of Medical Research for Autism Speaks and as the Vice President of Clinical Development and Medical Affairs at Neuren Pharmaceuticals. Currently, Dr. Horrigan serves as Chief Medical Officer for AMO Pharma Limited and is an Associate Consulting Professor for the Department of Psychiatry and Behavioral Sciences at Duke University School of Medicine.

As a clinician and investigator in studies at the University of North Carolina at Chapel Hill’s Developmental Neuropharmacology Clinic, many of Dr. Horrigan’s contributions went on to be approved medicines for children with neuropsychiatric disorders. Beyond symptom treatment, Dr. Horrigan has worked with pharmaceutical companies to create clinical programs for Rett syndrome, Fragile X and Angelman Syndrome. He was instrumental in helping launch Neuren’s clinical trials for NZ2566, known today as trofinetide.

In his career, Dr. Horrigan has seen over 100 patients with Rett syndrome. “I still remember well the very first girl I met with Rett syndrome,” said Dr. Horrigan. He shared that his patients have left an indelible impression and it drives him to spend his, “Energy, wits and tenacity to work hard, and I may be able to do some good.” Dr. Horrigan continued, “I’m very blessed to be able to do this work as my vocation—I wouldn’t want to do anything else. It’s why I exist.”

Dr. Horrigan’s vast expertise and passion for a cure, make him a strong asset not just to but to the entire Rett community. Rett syndrome is a rare disease caused by a genetic mutation that affects 1 in 10,000 females and is even more rare in males. There is no current treatment or cure, though many clinical trials are underway.

Executive Director of, Melissa Kennedy said, “Dr. Horrigan’s experience treating individuals with Rett syndrome and developing clinical trials and FDA-approved drugs adds significant momentum to our goal to end Rett syndrome.”

There has never been more momentum surrounding the advancement in research for Rett syndrome and Dr. Horrigan’s vision of what leading studies could yield is filled with hope. “The quality and volume of Rett syndrome research is now far ahead of other disorders. From a regulatory perspective, there is a great empathy for these research efforts in Rett syndrome to succeed. In the way of clinical trials and measures, things are becoming much more sophisticated.” He believes there is significant progress happening with genetics in regard to awakening the silent X to coming up with genetic remedies to creating a patch that will allow for normal protein function. He also noted, “There are many scientific and therapeutic advances happening adjacent to Rett syndrome that the Rett community can take advantage of.”

Dr. Horrigan shared that, “Working with [] is a premier opportunity to potentially have a broader impact than just helping the affected individual that is sitting in front of me.” This addition to the Board of Directors is far more than an advancement for one organization, it’s a victory for every individual and family in the fight to cure Rett syndrome.

About (The International Rett Syndrome Foundation) is one of the leading private funders of Rett syndrome research, investing over $48 million to date. The mission of the organization is to accelerate full spectrum research to cure Rett syndrome and empower families with information, knowledge and connectivity. recently earned Charity Navigator’s prestigious 4-star rating for its strong financial health and commitment to accountability and transparency. Further information about can be found at: