Monday January 29, 2024 12:30 PM US EST
Mary will share her son Henry's case and describe her efforts to develop an n=1 ASO for
him. Nina will contextualize ASO development within the wider scope of the AHC research
program, and discuss strategy for patient foundation investment across a portfolio of
therapeutics-focused studies. Mary and Nina will highlight the advantages of advancing both
N=1 and AHC community studies jointly, collaboratively, and in parallel, as partners with
researchers. Dr. Cat Lutz, Vice President of the Rare Disease Translational Center at the
Jackson Laboratory, will share details of her team's efforts to establish proof of concept for
AHC therapeutics in mouse models of AHC.
Mom of Annabel (and Clara)
President, Hope for Annabel
Nina leads the research program for Alternating Hemiplegia of Childhood (AHC). Nina has
worked to centralize, design, and oversee a portfolio of therapy-centric research efforts on
behalf of the AHC patient community.
Nina and Simon Frost founded Hope for Annabel in 2018 to help catalyze research for
therapies for AHC, and to serve as a research hub for the community. Hope for Annabel
works closely with other national AHC organizations, and maintains close ties to researchers
in the field.
Nina has led Hope for Annabel’s fundraising efforts since 2018. Nina also led Hope for
Annabel’s successful grant application for the $2 million Chan Zuckerberg Initiative award in
Nina’s prior professional experience was in management consulting. Nina and Simon met at
Cambridge University, where they were graduate students together. They now have two daughters,
Clara (10) and Annabel (7). Nina now works full time on AHC.
Dr. Cat Lutz, Ph.D
As the Vice President of the Rare Disease Translational Center (RDTC) at The Jackson
Laboratory (JAX), Dr Lutz works with patient-based foundations, researchers, and industry
partners to roadmap therapeutic strategies for rare diseases. Her work involves the engineering of
preclinical mouse models that represent patient-based mutations and the subsequent phenotyping
of the mice to explore clinically relevant, translatable phenotypes.
President, For Henry AHC
VP Research, Cure AHC
Mary is a member of the research team in the AHC community and leads research for the
development of an ASO for her son Henry.
Mary and her husband Anthony founded For Henry AHC in 2022 with the goal of raising
funds to advance a treatment for Henry with a current focus on ASO development. For
Henry AHC has raised $1.5M towards this effort to date.
Mary works closely with our national AHC foundations to build relationships with leading
experts and industry partners and to support research projects. Mary shares our research
team’s commitment to supporting our AHC community at large.
Before holding her most esteemed title of being Henry’s mom, Mary was a high school
English teacher and assistant principal. Mary holds a Master’s degree in education and
leadership from Stanford University and is a former Fulbrighter. Mary has embraced the
pivot from championing equity in education to pursuing therapies for AHC and works full time
on this endeavor and in caring for Henry, who is now 4.