Research Update | 8:00 a.m. – 12:00 p.m.

• Welcome – Theresa Pauca, Vice President, PHRF

• A Foundation’s Journey through Drug Development: The Need to Move FAST – Allyson Berent, DVM, Dipl. ACVIM, CSO, Foundation for Angelman Syndrome Therapeutics, COO, GeneTx Biotherapeutics, Director Interventional Endoscopy Services, The Animal Medical Center, Mother to Quincy: A beautiful little girl living with Angelman syndrome

• Pitt Hopkins 101 – An overview of the Therapeutic Landscape for Pitt Hopkins Syndrome – Audrey Davidow Lapidus, President, PHRF

• Gene Therapy From Concept To Creation:

Step 1 – Proving gene therapy works in Brain Organoid Models, Alysson Muotri, PhD – Virtual, University of California San Diego, School of Medicine

Step 2 – Taking the findings into mouse models, Patricia Cogram, PhD – Virtual, Associate Professor, Universidad de Chile 

Step 3 – Next Steps, From mouse model to human, Yael Weiss, CEO Mahzi Therapeutics

Break

• NNZ-2591 as a Treatment for Pitt Hopkins Syndrome – Nancy Jones, PhD, Vice President, Clinical Development, Neuren Pharmaceuticals

• Treatment options from the Colorado Clinic – Jessica Duis, MD, Associate Professor of Pediatrics & Medical Genetics, Director of Pitt-Hopkins Center of Excellence at Children’s Hospital Colorado

• Microbiota Transfer Therapy for Children with Pitt Hopkins Syndrome – James B. Adams, PhD and Rosa Krajmalnik-Brown, PhD, Arizona State University 

• Moving Forward – A Roadmap to Treatment – Audrey Davidow Lapidus, President, PHRF

Lunch in the atrium

Family Topics |  1:00 p.m. – 5:30 p.m. 

• Mom Break-Out Group* lead by Diane Krell, PTHS Mom and Director, PHRF

• Dad Break-Out Group* lead by Martin Cuevas, PTHS Dad and Clinical Coordinator

*Please note: Dad & Mom Breakout Sessions are strictly for parents (no children), so please plan accordingly. 

• From Overwhelmed to Empowered: A Journey Towards “Peace of Mind” and a 3-Month Solo Vacation – Ingrid Harding, PTHS Mom and Co-founder of Rett syndrome Research Trust and Girl Power 2 Cure

• Transition to Adulthood Panel Lead by PTHS moms Nicole Dyehouse, Ingrid Harding & Connie McNay

• Group Photo in the Atrium of the hotel 

Guests of the Embassy Suites have a complimentary evening reception available from 

5:00 – 6:30 p.m. 

Friday evening will be a night on your own, where you can decide what you would like to do but we recommend checking out Parkway Bank Park at Rosemont. Less than a 5 minute walk from our hotel, there is an assortment of food options (BBQ, Mexican, an Irish Pub, German Brewery and more) and entertainment for everyone (live music in the park, bowling alley, movie theater, iFLY indoor sky diving, & a dueling piano bar). We imagine our Pitt families will be taking over The Park Friday evening!

• 8:00 p.m. Viewing of Growing Together, a disability documentary by Sofia Pauca, Pitt Hopkins Sibling